Gene therapy for X-linked myotubular myopathy: the challenges.

Voermans, Nicol C; Ferreiro, Ana; Aartsema-Rus, Annemieke; Jungbluth, Heinz

Voermans, Nicol C; Ferreiro, Ana; Aartsema-Rus, Annemieke; Jungbluth, Heinz.

Afiliación

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 en En | MEDLINE | ID: mdl-37977700

Asunto(s)

Miopatías Estructurales Congénitas; Humanos; Miopatías Estructurales Congénitas/genética; Miopatías Estructurales Congénitas/terapia; Terapia Genética

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Miopatías Estructurales Congénitas Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article

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