GeneticsbioRxivPreprint
High-level correction of the sickle mutation amplified in vivo during erythroid differentiation
Wendy Magis, Mark A. DeWitt, Stacia K. Wyman…·David I. K. Martin
Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells achieves curative levels of editing.
17 citations