AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial.
作者信息
Baranello Giovanni, Muntoni Francesco
机构信息
The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, 30 Guilford Street, London, UK.
The Genetic Therapy Accelerator, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.
出版信息
Gene Ther. 2024 Nov;31(11-12):541-543. doi: 10.1038/s41434-024-00494-6. Epub 2024 Oct 23.
PMID:39443734
Abstract
摘要
Zotero 插件