Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness.

ABSTRACT: Genetic variants account for approximately half the cases of congenital and early-onset deafness. Methods and technologies for viral delivery of genes into the inner ear have evolved over the past decade to render gene therapy a viable and attractive approach for treatment. Variants in SYNE4, encoding the protein nesprin-4, a member of the linker of nucleoskeleton and cytoskeleton (LINC), lead to DFNB76 human deafness. Syne4^-/- mice have severe-to-profound progressive hearing loss and exhibit mislocalization of hair cell nuclei and hair cell degeneration. We used AAV9-PHP.B, a recently developed synthetic adeno-associated virus, to deliver the coding sequence of Syne4 into the inner ears of neonatal Syne4^-/- mice. Here we report rescue of hair cell morphology and surv

SUBMITTER: Taiber S 

PROVIDER: S-EPMC7863404 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature