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GJB2 gene therapy and conditional deletion reveal developmental stage-dependent effects on inner ear structure and function

Pathogenic variants in GJB2, the gene encoding connexin 26, are the most common cause of autosomal-recessive hereditary deafness. Despite this high prevalence, pathogenic mechanisms leading to GJB2-related deafness are not well understood, and cures are absent. Humans with GJB2-related deafness reta...

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Detalles Bibliográficos
Autores principales: Guo, Jingying, Ma, Xiaobo, Skidmore, Jennifer M., Cimerman, Jelka, Prieskorn, Diane M., Beyer, Lisa A., Swiderski, Donald L., Dolan, David F., Martin, Donna M., Raphael, Yehoash
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8531464/
https://www.ncbi.nlm.nih.gov/pubmed/34729379
http://dx.doi.org/10.1016/j.omtm.2021.09.009