Dual AAV gene therapy achieves recovery of hearing and auditory processing in a DFNB16 mouse model.

ABSTRACT:

Background

DFNB16, the second most common genetic cause of hearing loss, is caused by mutations of the STRC gene encoding stereocilin, a protein essential for the effective functioning of outer hair cells (OHCs) as cochlear amplifiers. Strc^-/- mice, which lack stereocilin, display severe to profound deafness and constitute a relevant preclinical model for DFNB16.

Methods

Using Strc^-/^- mice, we developed a gene therapy strategy based on the use of dual AAV9-PHP.eB vectors to deliver the full-length Strc cDNA. Therapeutic efficacy was assessed by evaluating stereocilin expression, OHC bundle architecture, and their attachment to the tectorial membrane, together with functional recovery using distortion product otoacoustic emissions (DPOAEs), au

SUBMITTER: Iranfar S 

PROVIDER: S-EPMC12784207 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature