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Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic option, but is limited by a potentially short therapeutic window and the constrained packaging capacity of the vector. We focus...

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Detalles Bibliográficos
Autores principales:	Akil, Omar, Dyka, Frank, Calvet, Charlotte, Emptoz, Alice, Lahlou, Ghizlene, Nouaille, Sylvie, Boutet de Monvel, Jacques, Hardelin, Jean-Pierre, Hauswirth, William W., Avan, Paul, Petit, Christine, Safieddine, Saaid, Lustig, Lawrence R.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	National Academy of Sciences 2019
Materias:	Biological Sciences
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6410774/ https://www.ncbi.nlm.nih.gov/pubmed/30782832 http://dx.doi.org/10.1073/pnas.1817537116

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