Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease affecting 1:5000 newborn males. No cure is currently available, but gene addition therapy, based on the adeno-associated viral (AAV) vector-mediated delivery of microdystrophin transgenes, is currently being tested in clinical trials....

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Detalles Bibliográficos
Autores principales: Cernisova, Viktorija, Lu-Nguyen, Ngoc, Trundle, Jessica, Herath, Shan, Malerba, Alberto, Popplewell, Linda
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10179398/
https://www.ncbi.nlm.nih.gov/pubmed/37175881
http://dx.doi.org/10.3390/ijms24098174

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10179398/
https://www.ncbi.nlm.nih.gov/pubmed/37175881
http://dx.doi.org/10.3390/ijms24098174

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