肾上腺脑白质营养不良
癌症
遗传增强
医学
基因
肿瘤科
遗传学
内科学
生物
过氧化物酶体
作者
Christine Duncan,Jacob R. Bledsoe,Bartosz Grzywacz,Amy Beckman,Melissa Bonner,Florian Eichler,Jörn‐Sven Kühl,Marian H. Harris,Sarah Slauson,Richard A. Colvin,Vinod K. Prasad,Gerald Downey,Francis J. Pierciey,Melissa A. Kinney,Marianna Foos,Ankit Lodaya,Nicole Floro,Geoffrey Parsons,Andrew C. Dietz,Ashish O. Gupta
标识
DOI:10.1056/nejmoa2405541
摘要
Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing
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