Outcomes of Gene Therapy for Severe Sickle Cell Disease (SCD) via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo With a Lentiviral ßAT87Q-Globin Vector

Biology of Blood and Marrow Transplantation - Netherlands
doi 10.1016/j.bbmt.2015.11.357

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