Therapeutic Application of Extracellular Vesicles-Capsulated Adeno-Associated Virus Vector via <i>nSMase2/Smpd3</i>, 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<sup>2+</sup> influx, resulting in protease activation, mitochondrial dysfu...

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Bibliographic Details
Main Authors: Yasunari Matsuzaka, Yukihiko Hirai, Kazuo Hashido, Takashi Okada
Format: Article
Language:English
Published: MDPI AG 2022-01-01
Series:International Journal of Molecular Sciences
Subjects:
Online Access:https://www.mdpi.com/1422-0067/23/3/1551