Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription

Abstract Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein. Adeno-associated virus (AAV)-based therapies aiming to restore dystrophin in muscles, by either exon skipping o...

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Bibliographic Details
Main Authors: Amédée Mollard, Cécile Peccate, Anne Forand, Julie Chassagne, Laura Julien, Pierre Meunier, Zoheir Guesmia, Thibaut Marais, Marc Bitoun, France Piétri-Rouxel, Sofia Benkhelifa-Ziyyat, Stéphanie Lorain
Format: Article
Language:English
Published: Nature Portfolio 2022-06-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-022-13405-9