Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in a mouse model of human Jervell and Lange‐Nielsen deafness syndrome
Abstract Mutations in the potassium channel subunit KCNQ1 cause the human severe congenital deafness Jervell and Lange‐Nielsen (JLN) syndrome. We applied a gene therapy approach in a mouse model of JLN syndrome (Kcnq1−/− mice) to prevent the development of deafness in the adult stage. A modified ade...
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| Główni autorzy: | , , , , , , , |
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| Format: | Artigo |
| Język: | Inglês |
| Wydane: |
Springer Nature
2015-06-01
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| Seria: | EMBO Molecular Medicine |
| Hasła przedmiotowe: | |
| Dostęp online: | https://doi.org/10.15252/emmm.201404929 |
| Etykiety: |
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