PMID- 36400545
OWN - NLM
STAT- MEDLINE
DCOM- 20221122
LR  - 20231202
IS  - 1558-1977 (Electronic)
IS  - 0889-8588 (Print)
IS  - 0889-8588 (Linking)
VI  - 36
IP  - 6
DP  - 2022 Dec
TI  - Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
PG  - 1313-1335
LID - S0889-8588(22)00065-X [pii]
LID - 10.1016/j.hoc.2022.06.007 [doi]
AB  - Curative therapies for sickle cell disease (SCD) include allogeneic human 
      leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell 
      transplant (HCT), gene therapy, and gene editing. However, comparative trial data 
      that might facilitate selecting one curative therapy over another are 
      unavailable. New strategies to decrease graft rejection and graft-versus-host 
      disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene 
      therapy and gene editing also has limitations. Herein, we review recent studies 
      on curative therapies for SCD in the past 5 years.
CI  - Published by Elsevier Inc.
FAU - Lawal, R AdeBisi
AU  - Lawal RA
AD  - Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood 
      Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 
      4-5140, Bethesda, MD 20892, USA; Hematology Branch, National Heart Lung and Blood 
      Institute, National Institutes of Health, Bethesda, MD, USA.
FAU - Walters, Mark C
AU  - Walters MC
AD  - University of California San Francisco Benioff Children's Hospital, 747 52nd 
      Street, Oakland CA 94609, USA.
FAU - Fitzhugh, Courtney D
AU  - Fitzhugh CD
AD  - Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood 
      Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 
      6N240A, Bethesda, MD 20892, USA. Electronic address: fitzhughc@nhlbi.nih.gov.
LA  - eng
GR  - U01 HL156620/HL/NHLBI NIH HHS/United States
GR  - Z99 HL999999/ImNIH/Intramural NIH HHS/United States
GR  - ZIA HL006211/ImNIH/Intramural NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, N.I.H., Intramural
PT  - Review
PL  - United States
TA  - Hematol Oncol Clin North Am
JT  - Hematology/oncology clinics of North America
JID - 8709473
SB  - IM
MH  - Humans
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Transplantation Conditioning
MH  - *Graft vs Host Disease/etiology/prevention & control
MH  - *Anemia, Sickle Cell/genetics/therapy
MH  - Genetic Therapy
MH  - Allografts
PMC - PMC9681017
MID - NIHMS1821267
OTO - NOTNLM
OT  - Allogeneic transplant
OT  - Gene editing
OT  - Gene therapy
OT  - Graft versus host disease
OT  - Haploidentical
OT  - Hematopoietic cell transplant
OT  - Matched sibling donor
OT  - Sickle cell disease
COIS- Disclosure M.C. Walters has consulting agreements with All Cells, Inc; BioChip 
      Labs, Inc; Ensoma, Inc; and Vertex Pharmaceuticals.
EDAT- 2022/11/19 06:00
MHDA- 2022/11/23 06:00
PMCR- 2023/12/01
CRDT- 2022/11/18 21:07
PHST- 2022/11/18 21:07 [entrez]
PHST- 2022/11/19 06:00 [pubmed]
PHST- 2022/11/23 06:00 [medline]
PHST- 2023/12/01 00:00 [pmc-release]
AID - S0889-8588(22)00065-X [pii]
AID - 10.1016/j.hoc.2022.06.007 [doi]
PST - ppublish
SO  - Hematol Oncol Clin North Am. 2022 Dec;36(6):1313-1335. doi: 
      10.1016/j.hoc.2022.06.007.