PMID- 23653154 OWN - NLM STAT- PubMed-not-MEDLINE DCOM- 20130509 LR - 20220809 IS - 2162-2531 (Print) IS - 2162-2531 (Electronic) IS - 2162-2531 (Linking) VI - 2 IP - 5 DP - 2013 May 7 TI - Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells. PG - e90 LID - 10.1038/mtna.2013.17 [doi] AB - Gene transfer into hCD34(+) hematopoietic stem/progenitor cells (HSCs) using human immunodeficiency virus type 1 (HIV-1)-based lentiviral vectors (LVs) has several promising therapeutic applications. Yet, efficiency, safety, and cost of LV gene therapy could be ameliorated by enhancing target cell transduction levels and reducing the amount of LV used on the cells. Several transduction enhancers already exist such as fibronectin fragments and cationic compounds, but all present limitations. In this study, we describe a new transduction enhancer called Vectofusin-1, which is a short cationic peptide, active on several LV pseudotypes. Vectofusin-1 is used as a soluble additive to safely increase the frequency of transduced HSCs and to augment the level of transduction to one or two copies of vector per cell in a vector dose-dependent manner. Vectofusin-1 acts at the entry step by promoting the adhesion and the fusion between viral and cellular membranes. Vectofusin-1 is therefore a promising additive that could significantly ameliorate hCD34(+) cell-based gene therapy.Molecular Therapy-Nucleic Acids (2013) 2, e90; doi:10.1038/mtna.2013.17; published online 7 May 2013. FAU - Fenard, David AU - Fenard D AD - 1] Genethon, Evry, France [2] INSERM, UMR_S951, Genethon, Evry, France [3] Universite Evry Val d'Essonne, UMR_S951, Evry, France. FAU - Ingrao, Dina AU - Ingrao D FAU - Seye, Ababacar AU - Seye A FAU - Buisset, Julien AU - Buisset J FAU - Genries, Sandrine AU - Genries S FAU - Martin, Samia AU - Martin S FAU - Kichler, Antoine AU - Kichler A FAU - Galy, Anne AU - Galy A LA - eng PT - Journal Article DEP - 20130507 PL - United States TA - Mol Ther Nucleic Acids JT - Molecular therapy. Nucleic acids JID - 101581621 PMC - PMC4817938 EDAT- 2013/05/09 06:00 MHDA- 2013/05/09 06:01 PMCR- 2013/05/01 CRDT- 2013/05/09 06:00 PHST- 2013/05/09 06:00 [entrez] PHST- 2013/05/09 06:00 [pubmed] PHST- 2013/05/09 06:01 [medline] PHST- 2013/05/01 00:00 [pmc-release] AID - S2162-2531(16)30149-4 [pii] AID - 10.1038/mtna.2013.17 [doi] PST - epublish SO - Mol Ther Nucleic Acids. 2013 May 7;2(5):e90. doi: 10.1038/mtna.2013.17.