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...
Main Authors: | , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2022-06-01
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Series: | Scientific Reports |
Online Access: | https://doi.org/10.1038/s41598-022-13405-9 |