Published Paper Details:

Sensorineural hearing loss (SNHL) is the most common form of permanent auditory impairment, affecting millions worldwide and profoundly influencing communication, cognition, and quality of life. Conventional approaches, such as hearing aids and cochlear implants, provide compensation but cannot restore the physiological processes underlying natural hearing. Recent advances in molecular medicine have positioned gene therapy as a transformative strategy for hereditary and acquired forms of SNHL. This review examines current progress in gene therapy for otology, emphasizing three primary mechanisms: gene replacement, gene editing, and regenerative or neuroprotective strategies. Clinical trials targeting OTOF mutations using dual-adeno-associated viral (AAV) vectors have demonstrated measurable restoration of hearing in infants and children with auditory neuropathy, marking a milestone in translational medicine. Preclinical progress with GJB2 replacement and early trials of ATOH1-based regenerative approaches further highlight the diversity of therapeutic targets. Nonetheless, challenges remain, including vector capacity limits, immune barriers to re-dose, narrow developmental windows of efficacy, and global accessibility concerns. Future directions point toward improved vector engineering, precise genome-editing technologies for dominant mutations, integration of gene therapy with cochlear implants, and expansion of treatment to broader patient populations. Collectively, the field is moving from experimental feasibility toward clinical reality, offering hope that gene therapy may soon provide durable, biologically restorative solutions for individuals affected by hearing loss