AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy

In the context of future adeno-associated viral (AAV)–based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We showed that AAV genomes encoding non-therapeutic U7 were lost...

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Detalhes bibliográficos
Main Authors: Le Hir, Maëva, Goyenvalle, Aurélie, Peccate, Cécile, Précigout, Guillaume, Davies, Kay E, Voit, Thomas, Garcia, Luis, Lorain, Stéphanie
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3734654/
https://ncbi.nlm.nih.gov/pubmed/23752313
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.121
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