High-level correction of the sickle mutation amplified in vivo during erythroid differentiation
Wendy Magis, Mark A. DeWitt, Stacia K. Wyman, Jonathan T. Vu, Seok‐Jin Heo, Shirley J. Shao, Fiona Hennig, Zulema G. Romero, Beatriz Campo-Fernández, Matthew McNeill, Garrett R. Rettig, Yongming Andrew Sun, Patrick J. Lau, Yu Wang, Mark A. Behlke, , David I. K. Martin
Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells achieves curative levels of editing.
bioRxiv · Jan 8, 2019 · PreprintGenetics