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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Autores principales:	Qing Chang, Jianjun Wang, Qi Li, Yeunjung Kim, Binfei Zhou, Yunfeng Wang, Huawei Li, Xi Lin
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Springer Nature 2015-06-01
Colección:	EMBO Molecular Medicine
Materias:	gene therapy hearing restoration Jervell and Lange‐Nielsen syndrome Kcnq1 null mice virus
Acceso en línea:	https://doi.org/10.15252/emmm.201404929
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