Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction.

DeWitt M, Campo-Fernandez B, Roy S, Kleinhenz ALW, Davila A, Lennon M, Vu JT, Krishnappa N, Said S, Tseng CH, Park SH, Bao G, Magis W, Martin DIK, Boffelli D, Wyman SK, Kohn DB, Walters MC, Romero Z