CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

• DOI: 10.1126/scitranslmed.aah3480
• Publication Date: 2017-01-11T19:15:44Z
• Authors: Suk See De Ravin; Linhong Li; Xiaolin Wu; Uimook Choi; Cornell Allen; Sherry Koontz; Janet Lee; Narda Theobald-Wh...; Jessica Chu; Mary Garofalo; Colin Sweeney; Lela Kardava; Susan Moir; Angelia Viley; Pachai Natarajan; Ling Su; Douglas Kuhns; Kol A. Zarember; Madhusudan V. Peshwa; Harry L. Malech
• Keywords: Chronic Granulomatous Disease

CRISPR-mediated gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease resulted in functional human leukocytes mice after transplantation.