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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Udgivet i:EMBO Mol Med
Main Authors: Taiber, Shahar, Cohen, Roie, Yizhar‐Barnea, Ofer, Sprinzak, David, Holt, Jeffrey R, Avraham, Karen B
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
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Articles
Online adgang: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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