PMID- 34788506
OWN - NLM
STAT- MEDLINE
DCOM- 20211203
LR  - 20211214
IS  - 1533-4406 (Electronic)
IS  - 0028-4793 (Linking)
VI  - 385
IP  - 21
DP  - 2021 Nov 18
TI  - Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
PG  - 1929-1940
LID - 10.1056/NEJMoa2106596 [doi]
AB  - BACKGROUND: Allogeneic hematopoietic stem-cell transplantation is the standard of 
      care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). 
      However, this treatment is only partially curative and is associated with 
      complications. METHODS: We are conducting an ongoing study involving eight 
      children with MPSIH. At enrollment, the children lacked a suitable allogeneic 
      donor and had a Developmental Quotient or Intelligence Quotient score above 70 
      (i.e., none had moderate or severe cognitive impairment). The children received 
      autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo 
      with an alpha-L-iduronidase (IDUA)-encoding lentiviral vector after myeloablative 
      conditioning. Safety and correction of blood IDUA activity up to supraphysiologic 
      levels were the primary end points. Clearance of lysosomal storage material as 
      well as skeletal and neurophysiological development were assessed as secondary 
      and exploratory end points. The planned duration of the study is 5 years. 
      RESULTS: We now report interim results. The children's mean (+/-SD) age at the time 
      of HSPC gene therapy was 1.9+/-0.5 years. At a median follow-up of 2.10 years, the 
      procedure had a safety profile similar to that known for autologous hematopoietic 
      stem-cell transplantation. All the patients showed prompt and sustained 
      engraftment of gene-corrected cells and had supraphysiologic blood IDUA activity 
      within a month, which was maintained up to the latest follow-up. Urinary 
      glycosaminoglycan (GAG) excretion decreased steeply, reaching normal levels at 12 
      months in four of five patients who could be evaluated. Previously undetectable 
      levels of IDUA activity in the cerebrospinal fluid became detectable after gene 
      therapy and were associated with local clearance of GAGs. Patients showed stable 
      cognitive performance, stable motor skills corresponding to continued motor 
      development, improved or stable findings on magnetic resonance imaging of the 
      brain and spine, reduced joint stiffness, and normal growth in line with World 
      Health Organization growth charts. CONCLUSIONS: The delivery of HSPC gene therapy 
      in patients with MPSIH resulted in extensive metabolic correction in peripheral 
      tissues and the central nervous system. (Funded by Fondazione Telethon and 
      others; ClinicalTrials.gov number, NCT03488394; EudraCT number, 2017-002430-23.).
CI  - Copyright (c) 2021 Massachusetts Medical Society.
FAU - Gentner, Bernhard
AU  - Gentner B
AUID- ORCID: 0000-0001-6024-4718
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Tucci, Francesca
AU  - Tucci F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Galimberti, Stefania
AU  - Galimberti S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Fumagalli, Francesca
AU  - Fumagalli F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - De Pellegrin, Maurizio
AU  - De Pellegrin M
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Silvani, Paolo
AU  - Silvani P
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Camesasca, Chiara
AU  - Camesasca C
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Pontesilli, Silvia
AU  - Pontesilli S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Darin, Silvia
AU  - Darin S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Ciotti, Francesca
AU  - Ciotti F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Sarzana, Marina
AU  - Sarzana M
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Consiglieri, Giulia
AU  - Consiglieri G
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Filisetti, Chiara
AU  - Filisetti C
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Forni, Giulia
AU  - Forni G
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Passerini, Laura
AU  - Passerini L
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Tomasoni, Daniela
AU  - Tomasoni D
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Cesana, Daniela
AU  - Cesana D
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Calabria, Andrea
AU  - Calabria A
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Spinozzi, Giulio
AU  - Spinozzi G
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Cicalese, Maria-Pia
AU  - Cicalese MP
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Calbi, Valeria
AU  - Calbi V
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Migliavacca, Maddalena
AU  - Migliavacca M
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Barzaghi, Federica
AU  - Barzaghi F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Ferrua, Francesca
AU  - Ferrua F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Gallo, Vera
AU  - Gallo V
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Miglietta, Simona
AU  - Miglietta S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Zonari, Erika
AU  - Zonari E
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Cheruku, Patali S
AU  - Cheruku PS
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Forni, Claudia
AU  - Forni C
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Facchini, Marcella
AU  - Facchini M
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Corti, Ambra
AU  - Corti A
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Gabaldo, Michela
AU  - Gabaldo M
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Zancan, Stefano
AU  - Zancan S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Gasperini, Serena
AU  - Gasperini S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Rovelli, Attilio
AU  - Rovelli A
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Boelens, Jaap-Jan
AU  - Boelens JJ
AUID- ORCID: 0000-0003-2232-6952
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Jones, Simon A
AU  - Jones SA
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Wynn, Robert
AU  - Wynn R
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Baldoli, Cristina
AU  - Baldoli C
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Montini, Eugenio
AU  - Montini E
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Gregori, Silvia
AU  - Gregori S
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Ciceri, Fabio
AU  - Ciceri F
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Valsecchi, Maria G
AU  - Valsecchi MG
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - la Marca, Giancarlo
AU  - la Marca G
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Parini, Rossella
AU  - Parini R
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Naldini, Luigi
AU  - Naldini L
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Aiuti, Alessandro
AU  - Aiuti A
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
FAU - Bernardo, Maria-Ester
AU  - Bernardo ME
AD  - From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. 
      Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. 
      Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., 
      F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), 
      Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric 
      Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon 
      Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., 
      G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., 
      E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific 
      Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), 
      and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of 
      Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), 
      Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of 
      Experimental and Clinical Biomedical Sciences, University of Florence, and 
      Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's 
      University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, 
      Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, 
      Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and 
      Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York 
      (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. 
      Mary's Hospital, Manchester University NHS Foundation Trust, University of 
      Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, 
      Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
CN  - MPSI Study Group
LA  - eng
SI  - ClinicalTrials.gov/NCT03488394
SI  - EudraCT/2017-002430-23
PT  - Clinical Trial, Phase I
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - N Engl J Med
JT  - The New England journal of medicine
JID - 0255562
RN  - 0 (Glycosaminoglycans)
RN  - EC 3.2.1.76 (IDUA protein, human)
RN  - EC 3.2.1.76 (Iduronidase)
SB  - IM
CIN - N Engl J Med. 2021 Nov 18;385(21):2003-2004. PMID: 34788512
MH  - Child, Preschool
MH  - Female
MH  - Follow-Up Studies
MH  - *Genetic Therapy
MH  - Genetic Vectors
MH  - Glycosaminoglycans/urine
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Iduronidase/deficiency/genetics/*metabolism
MH  - Infant
MH  - Lentivirus
MH  - Male
MH  - Mucopolysaccharidosis I/metabolism/*therapy
MH  - Mutation
MH  - Stem Cell Transplantation
MH  - Transplantation, Autologous
FIR - Basilico, Federica
IR  - Basilico F
FIR - Bykova, Tatiana
IR  - Bykova T
FIR - Calvi, Maria Rosa
IR  - Calvi MR
FIR - Castelle, Martin
IR  - Castelle M
FIR - Bona, Maria Della
IR  - Bona MD
FIR - Elenga, Narcisse
IR  - Elenga N
FIR - De Las Heras, Javier
IR  - De Las Heras J
FIR - Funghini, Silvia
IR  - Funghini S
FIR - Groselj, Urh
IR  - Groselj U
FIR - Malvagia, Sabrina
IR  - Malvagia S
FIR - Martin, Elise
IR  - Martin E
FIR - Mikhaylova, Svetlana
IR  - Mikhaylova S
FIR - Neven, Benedicte
IR  - Neven B
FIR - Njuieyon, Falucar
IR  - Njuieyon F
FIR - Ombrone, Daniela
IR  - Ombrone D
FIR - Serafini, Marta
IR  - Serafini M
FIR - Tal, Galit
IR  - Tal G
FIR - Visagalli, Ilaria
IR  - Visagalli I
EDAT- 2021/11/18 06:00
MHDA- 2021/12/15 06:00
CRDT- 2021/11/17 17:34
PHST- 2021/11/17 17:34 [entrez]
PHST- 2021/11/18 06:00 [pubmed]
PHST- 2021/12/15 06:00 [medline]
AID - 10.1056/NEJMoa2106596 [doi]
PST - ppublish
SO  - N Engl J Med. 2021 Nov 18;385(21):1929-1940. doi: 10.1056/NEJMoa2106596.