Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease

DB Kohn, C Booth, EM Kang. Nature Med. 2020;26(2):200–206 To assess the safety and evaluate the efficacy and stability of ex vivo autologous CD34+ hematopoietic stem and progenitor cell based lentiviral gene therapy for X-linked CGD. This study included 9 male patients with X-CGD (confirmed by DNA sequencing and absent NADPH-oxidase activity) who lacked a 10/10 HLA-matched donor. This is a multicenter prospective study to evaluate the safety and efficacy of lentiviral gene therapy for X-CGD. Patients’ clinical characteristics, laboratory findings …