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 BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells. 138 Molecular Therapy: Methods & Clinical Development Vol. 4 March 2017. doi:  10.1016/j.omtm.2016.12.009