作者
Florian Eichler,Christine Duncan,Patricia L. Musolino,Troy C. Lund,Ashish 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,Elizabeth McNeil,Laura A. Demopoulos,Andrew C. Dietz,Himal L. Thakar,Paul J. Orchard,David A. Williams
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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