Hereditary hearing loss constitutes a growing global health burden, with genetic factors accounting for 60% of congenital cases and over 200 related genes identified. Current treatments like cochlear implants cannot reverse inner ear damage. This review summarizes advances in gene therapy tools, strategies, and clinical progress for hereditary hearing loss, aiming to boost its translational development.
This review focuses on the classification and genetic characteristics of hereditary hearing loss, distinguishing non‑syndromic and syndromic subtypes with their respective pathogenic genes. It systematically introduces inner ear gene delivery platforms including optimized AAV vectors and cell‑specific promoters, and elaborates three core strategies: gene replacement, suppression, and precision editing. Additionally, it summarizes key preclinical advances in major deafness genes such as OTOF, GJB2, STRC, SLC26A4 and USH1C, and comprehensively outlines landmark clinical progress in OTOF‑targeted trials, reflecting the full translational chain of research.
This review establishes a standardized clinical paradigm for hereditary hearing loss gene therapy, clarifies key translational hurdles including vector limitations and safety issues, and provides a scientific roadmap to accelerate the development of safer, more precise, and durable treatments for patients worldwide. The work entitled “Gene Therapy for Hereditary Hearing Loss: Comprehensive Overview and Clinical Applications” was published on ENT Discovery (published on Apr. 01, 2026). Reference: Yixin Xu, Guannan Geng, Ziting Chen, Shuang Han, Shaowei Hu, Xintai Fan, Hui Wang, Huawei Li, Yilai Shu. Gene Therapy for Hereditary Hearing Loss: Comprehensive Overview and Clinical Applications. ENT Disc, 2026, 2(1): 7-21
DOI: 10.15302/ENTD.2026.030006