Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G

Our understanding of the mechanisms underlying inherited forms of inner ear deficits has considerably improved during the past 20 y, but we are still far from curative treatments. We investigated gene replacement as a strategy for restoring inner ear functions in a mouse model of Usher syndrome type...

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Bibliografiset tiedot
Julkaisussa:Proc Natl Acad Sci U S A
Päätekijät: Emptoz, Alice, Michel, Vincent, Lelli, Andrea, Akil, Omar, Boutet de Monvel, Jacques, Lahlou, Ghizlene, Meyer, Anaïs, Dupont, Typhaine, Nouaille, Sylvie, Ey, Elody, Franca de Barros, Filipa, Beraneck, Mathieu, Dulon, Didier, Hardelin, Jean-Pierre, Lustig, Lawrence, Avan, Paul, Petit, Christine, Safieddine, Saaid
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: National Academy of Sciences 2017
Aiheet:
Biological Sciences
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC5594693/
https://ncbi.nlm.nih.gov/pubmed/28835534
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1708894114
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