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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Veröffentlicht in:Proc Natl Acad Sci U S A
Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2019
Schlagworte:
Biological Sciences
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6410774/
https://ncbi.nlm.nih.gov/pubmed/30782832
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1817537116
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