Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Gene therapy and antisense approaches hold promise for the treatment of Duchenne muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. We previously reported the efficacy of AAV-U7 small...

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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Aupy, Philippine, Zarrouki, Faouzi, Sandro, Quentin, Gastaldi, Cécile, Buclez, Pierre-Olivier, Mamchaoui, Kamel, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2020
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7240049/
https://ncbi.nlm.nih.gov/pubmed/32462052
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.025
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