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...

Full description

Bibliographic Details
Main Authors: Jingying Guo, Xiaobo Ma, Jennifer M. Skidmore, Jelka Cimerman, Diane M. Prieskorn, Lisa A. Beyer, Donald L. Swiderski, David F. Dolan, Donna M. Martin, Yehoash Raphael
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001479