Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction.
Data support initiation of a first-in-human phase I trial of CRISPR_SCD001 for sickle patients presenting with severe pathology, and generates a clinical-grade, cryopreserved CD34+ cell product that corrects the sickle mutation, restores anti-sickling hemoglobins, and meets pre-clinical safety criteria.
M. DeWitt, Beatriz Campo-Fernández, Sohini Roy et al.
· Molecular Therapy · 0 citations