Update on Gene Therapy in the Treatment of Hereditary Hearing Loss

ABSTRACT Gene therapy is a promising therapeutic approach for genetic disorders, involving genetic modification to repair or reconstruct faulty genetic material. It is particularly relevant to hereditary hearing loss (HHL), a common monogenic condition that can lead to congenital deafness. The recent approval of clinical trial results using gene therapy for HHL underscores the growing interest in this field. To further advance inner ear gene therapy and its application in genetic diseases, it is crucial to review the progress of gene therapy for HHL. This review focuses on the three major gene therapy strategies—gene replacement, gene suppression, and gene editing—highlighting their application across different monogenic disorders and successful preclinical trials in HHL. We summarize the primary gene therapy strategies used in recent years, discuss recent achievements in preclinical studies, and explore potential advancements in this field.