Cell BiologybioRxivPreprint
Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein Complex
Mark A. DeWitt, Wendy Magis, Nicolas L. Bray…·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.
DOI 10.1101/036236