Ex Vivo Selection of Transduced Hematopoietic Stem Cells for Gene Therapy of β-Hemoglobinopathies

Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in recent trials for several individuals with inherited disorders, transduction incompleteness of the HSC population remains a hurdle to yield a cure for all patients with reasonably low integrated vector number...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Bhukhai, Kanit, de Dreuzy, Edouard, Giorgi, Marie, Colomb, Charlotte, Negre, Olivier, Denaro, Maria, Gillet-Legrand, Béatrix, Cheuzeville, Joëlle, Paulard, Anaïs, Trebeden-Negre, Hélène, Borwornpinyo, Suparerk, Sii-Felice, Karine, Maouche, Leila, Down, Julian D., Leboulch, Phillippe, Payen, Emmanuel
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5835017/
https://ncbi.nlm.nih.gov/pubmed/29221807
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.10.015
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