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Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness

Genetic variants account for approximately half the cases of congenital and early‐onset deafness. Methods and technologies for viral delivery of genes into the inner ear have evolved over the past decade to render gene therapy a viable and attractive approach for treatment. Variants in SYNE4, encodi...

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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: Taiber, Shahar, Cohen, Roie, Yizhar‐Barnea, Ofer, Sprinzak, David, Holt, Jeffrey R, Avraham, Karen B
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7863404/
https://ncbi.nlm.nih.gov/pubmed/33350593
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.202013259
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