MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

Duchenne muscular dystrophy (DMD) is caused by dystrophin deficiency resulting in progressive muscle weakness and fibrotic scarring. Muscle fibrosis impairs blood flow, hampering muscle repair and regeneration. Irrespective of the success of gene restoration, functional improvement is limited withou...

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Veröffentlicht in:JCI Insight
Hauptverfasser: Heller, Kristin N., Mendell, Joshua T., Mendell, Jerry R., Rodino-Klapac, Louise R.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2017
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5414552/
https://ncbi.nlm.nih.gov/pubmed/28469083
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.93309
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