A Novel High-Titer, Bifunctional Lentiviral Vector for Autologous Hematopoietic Stem Cell Gene Therapy of Sickle Cell Disease

Kevyn L. Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernández, Kevin Tam, Katherine Orr, R. Hollis, Christian Brendel, David A. Williams, Donald B. Kohn
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用于镰状细胞病自体造血干细胞基因治疗的新型高滴度双功能慢病毒载体
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