Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness
Abstract 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 SYNE...
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| Principais autores: | , , , , , |
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| 格式: | Artigo |
| 语言: | Inglês |
| 出版: |
Springer Nature
2020-12-01
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| 丛编: | EMBO Molecular Medicine |
| 主题: | |
| 在线阅读: | https://doi.org/10.15252/emmm.202013259 |
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