Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice

Recessive PJVK mutations that cause a deficiency of pejvakin, a protein expressed in both sensory hair cells and first-order neurons of the inner ear, are an important cause of hereditary hearing impairment. Patients with PJVK mutations garner limited benefits from cochlear implantation; thus, alter...

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Detalles Bibliográficos
Autores principales: Lu, Ying-Chang, Tsai, Yi-Hsiu, Chan, Yen-Huei, Hu, Chin-Ju, Huang, Chun-Ying, Xiao, Ru, Hsu, Chuan-Jen, Vandenberghe, Luk H., Wu, Chen-Chi, Cheng, Yen-Fu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9714786/
https://www.ncbi.nlm.nih.gov/pubmed/36278489
http://dx.doi.org/10.1172/jci.insight.152941

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