rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy

X-linked myotubular myopathy (XLMTM) is a life-threatening skeletal muscle disease caused by mutations in the MTM1 gene. XLMTM fibres display a population of nuclei mispositioned in the centre. In the present study, we aimed to explore whether positioning and overall distribution of nuclei affects c...

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Detalles Bibliográficos
Autores principales: Ross, Jacob A., Tasfaout, Hichem, Levy, Yotam, Morgan, Jennifer, Cowling, Belinda S., Laporte, Jocelyn, Zanoteli, Edmar, Romero, Norma B., Lowe, Dawn A., Jungbluth, Heinz, Lawlor, Michael W., Mack, David L., Ochala, Julien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7574461/
https://www.ncbi.nlm.nih.gov/pubmed/33076971
http://dx.doi.org/10.1186/s40478-020-01048-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7574461/
https://www.ncbi.nlm.nih.gov/pubmed/33076971
http://dx.doi.org/10.1186/s40478-020-01048-8

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