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Gene therapy for X-linked myotubular myopathy: the challenges.
Voermans, Nicol C; Ferreiro, Ana; Aartsema-Rus, Annemieke; Jungbluth, Heinz.
Affiliation
  • Voermans NC; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6500 HB Nijmegen, Netherlands. Electronic address: nicol.voermans@radboudumc.nl.
  • Ferreiro A; Basic and Translational Myology Laboratory, Université Paris Cité, BFA, CNRS UMR8251, Paris, France; Reference Centre for Neuromuscular Disorders, Institut of Myology, Neuromyology Department, Pitié-Salpêtrière Hospital, AP-HP, Paris, France.
  • Aartsema-Rus A; Department of Human Genetics, Leiden University Medical Center, Leiden, Netherlands.
  • Jungbluth H; Department of Paediatric Neurology, Neuromuscular Service, Evelina Children Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK; Randall Centre for Cell and Molecular Biophysics, Muscle Signalling Section, Faculty of Life Sciences and Medicine, King's College London, London, UK.
Lancet Neurol ; 22(12): 1089-1091, 2023 12.
Article in En | MEDLINE | ID: mdl-37977700

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Myopathies, Structural, Congenital Limits: Humans Language: En Journal: Lancet Neurol Journal subject: NEUROLOGIA Year: 2023 Document type: Article

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Myopathies, Structural, Congenital Limits: Humans Language: En Journal: Lancet Neurol Journal subject: NEUROLOGIA Year: 2023 Document type: Article