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Therapeutic Application of Extracellular Vesicles-Capsulated Adeno-Associated Virus Vector via nSMase2/Smpd3, Satellite, and Immune Cells in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is caused by loss-of-function mutations in the dystrophin gene on chromosome Xp21. Disruption of the dystrophin–glycoprotein complex (DGC) on the cell membrane causes cytosolic Ca(2+) influx, resulting in protease activation, mitochondrial dysfunction, and progressi...

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Detalles Bibliográficos
Autores principales: Matsuzaka, Yasunari, Hirai, Yukihiko, Hashido, Kazuo, Okada, Takashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8836108/
https://www.ncbi.nlm.nih.gov/pubmed/35163475
http://dx.doi.org/10.3390/ijms23031551