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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:EMBO Mol Med
Prif Awduron: Taiber, Shahar, Cohen, Roie, Yizhar‐Barnea, Ofer, Sprinzak, David, Holt, Jeffrey R, Avraham, Karen B
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: John Wiley and Sons Inc. 2020
Pynciau:
Articles
Mynediad Ar-lein: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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