Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

• DOI: 10.1126/scitranslmed.aaf9336
• Publication Date: 2016-10-12T18:18:14Z
• Authors: Mark A. DeWitt; Wendy Magis; Nicolas L. Bray; Tianjiao Wang; Jennifer R. Berman; Fabrizia Urbinati; Seok-Jin Heo; Therese Mitros; Denise P. Muñoz; Dario Boffelli; Donald B. Kohn; Mark C. Walters; Dana Carroll; David I. K. Martin; Jacob E. Corn
• Keywords: Hematopoietic stem cell

Hematopoietic stem cells from patients with sickle cell disease can be edited by CRISPR/Cas9 and maintain the edits in vivo.