Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease

Huang, Khan, Au, Barber, López-Vásquez, Prokopishyn, Boutin, Rothe, Rip, Abaoui, Nagree, Dworski, Schambach, Keating, West, Klassen, Turner, Sirrs, Rupar, Auray-Blais, Foley, Medin. Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease. Molecular Therapy: Methods & Clinical Development Vol. 5 June 2017. doi:  10.1016/j.omtm.2017.05.003

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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells

Kai-Hsin Chang, Sarah E. Smith, Timothy Sullivan, Kai Chen, Qianhe Zhou, Jason A. West, Mei Liu, Yingchun Liu, Benjamin F. Vieira, Chao Sun, Vu P. Hong, Mingxuan Zhang, Xiao Yang, Andreas Reik, Fyodor D. Urnov, Edward J. Rebar, Michael C. Holmes, Olivier Danos, Haiyan Jiang, and Siyuan Tan. Long-Term Engraftment and Fetal Globin Induction upon…

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