肾上腺脑白质营养不良
遗传增强
医学
病毒学
基因
神经科学
生物
遗传学
过氧化物酶体
作者
Florian Eichler,Christine Duncan,Patricia Musolino,Troy C. Lund,Ashish O. Gupta,Satiro De Oliveira,Adrian J. Thrasher,Patrick Aubourg,Jörn‐Sven Kühl,Daniel J. Loes,Hernán Amartino,Nicholas Smith,Juliana Folloni Fernandes,Caroline Sevin,Raman Sankar,Shaun A. Hussain,Paul Gissen,Jean‐Hugues Dalle,Uwe Platzbecker,Gerald Downey
标识
DOI：10.1056/nejmoa2400442
摘要
Cerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy characterized by white-matter disease, loss of neurologic function, and early death. Elivaldogene autotemcel (eli-cel) gene therapy, which consists of autologous CD34+ cells transduced with Lenti-D lentiviral vector containing
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