Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate

Hereditary hearing loss often results from mutation of genes expressed by cochlear hair cells. Gene addition using AAV vectors has shown some efficacy in mouse models, but clinical application requires two additional advances. First, new AAV capsids must mediate efficient transgene expression in bot...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Mol Ther Methods Clin Dev
Main Authors: György, Bence, Meijer, Elise J., Ivanchenko, Maryna V., Tenneson, Kelly, Emond, Frederick, Hanlon, Killian S., Indzhykulian, Artur A., Volak, Adrienn, Karavitaki, K. Domenica, Tamvakologos, Panos I., Vezina, Mark, Berezovskii, Vladimir K., Born, Richard T., O’Brien, Maureen, Lafond, Jean-François, Arsenijevic, Yvan, Kenna, Margaret A., Maguire, Casey A., Corey, David P.
פורמט: Artigo
שפה:Inglês
יצא לאור: American Society of Gene & Cell Therapy 2018
נושאים:
Article
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC6297893/
https://ncbi.nlm.nih.gov/pubmed/30581889
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.11.003
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