Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein Complex
Mark A. DeWitt, Wendy Magis, Nicolas L. Bray, Tianjiao Wang, Jennifer R. Berman, Fabrizia Urbinati, Denise P. Muñoz, Donald B. Kohn, Mark C. Walters, Dana Carroll, David K. Martin, Jacob E. Corn
This study corrects the sickle mutation in human hematopoietic stem cells using a Cas9 RNP/ssDNA donor approach, showing long-term therapeutic editing in mice.
bioRxiv · Jan 15, 2016 · PreprintCell Biology