Hematopoietic Stem-Cell Gene Therapy for Cystinosis
胱氨酸病的造血干细胞基因治疗
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2506431
Barshop, Bruce A; Ball, Edward D; Benador, Nadine; Trauner, Doris; Phillips, Susan; Dohil, Ranjan; Afshari, Natalie A; Roy, Sohini; Campo Fernandes, Beatriz; Kohn, Donald; Shayan, Katayoon; Everett, John K; Bushman, Frederic D; Midgley, Julian; Liang, Hong; Sawyers, Anne; Gangoiti, Jon A; Panchal, Maulik; Ahmed, Imama; Cherqui, Stephanie