Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy

Robust expression of shortened, functional dystrophin provided impetus to develop adeno-associated virus (AAV)–based constructs for clinical application. Because several cassettes are being tested in clinical trials, this study compared the efficacies of four shortened dystrophin-promoter combinatio...

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Detalles Bibliográficos
Autores principales: Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10538294/
https://www.ncbi.nlm.nih.gov/pubmed/37670674
http://dx.doi.org/10.1242/bio.059797

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10538294/
https://www.ncbi.nlm.nih.gov/pubmed/37670674
http://dx.doi.org/10.1242/bio.059797

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