Gene therapy for spinal muscular atrophy: timing is key.
作者信息
Servais Laurent
机构信息
MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford and NIHR Oxford Biomedical Research Center, Oxford OX3 9DU, United Kingdom.
Neuromuscular Center of Liège, Division of Paediatrics, CHU and University of Liège, 1 Bvd du XII de Ligne, Liège 4000, Belgium.
出版信息
Lancet Reg Health Eur. 2024 Oct 21;47:101112. doi: 10.1016/j.lanepe.2024.101112. eCollection 2024 Dec.
Abstract
摘要
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