Gene therapy for X-linked myotubular myopathy: the challenges

被引：4
|
作者：
Voermans, Nicol C. [1 ]
Ferreiro, Ana [2 ,3 ]
Aartsema-Rus, Annemieke [4 ]
Jungbluth, Heinz [5 ,6 ]
机构：
[1] Radboud Univ Nijmegen, Dept Neurol, Med Ctr, Donders Inst Brain Cognit & Behav, NL-6500 HB Nijmegen, Netherlands
[2] Univ Paris, CNRS UMR8251, Basic & Translat Myol Lab, Paris, France
[3] Hop La Pitie Salpetriere, AP HP, Reference Ctr Neuromuscular Disorders, Neuromyol Dept,Inst Myol, Paris, France
[4] Leiden Univ, Med Ctr, Dept Human Genet, Leiden, Netherlands
[5] Guys & St Thomas NHS Fdn Trust, Evelina Children Hosp, Dept Paediat Neurol, Neuromuscular Serv, London, England
[6] Kings Coll London, Fac Life Sci & Med, Randall Ctr Cell & Mol Biophys, Muscle Signalling Sect, London, England
来源：
LANCET NEUROLOGY | 2023年 / 22卷 / 12期
关键词：
D O I：
10.1016/S1474-4422(23)00416-7
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
引用
收藏
页码：1089 / 1091
页数：3
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