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

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 adeno-asso...

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Detalles Bibliográficos
Autores principales: Chang, Qing, Wang, Jianjun, Li, Qi, Kim, Yeunjung, Zhou, Binfei, Wang, Yunfeng, Li, Huawei, Lin, Xi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Ltd 2015
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4551345/
https://www.ncbi.nlm.nih.gov/pubmed/26084842
http://dx.doi.org/10.15252/emmm.201404929

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4551345/
https://www.ncbi.nlm.nih.gov/pubmed/26084842
http://dx.doi.org/10.15252/emmm.201404929

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