1. Department of Radiology, Zhuhai People’s Hospital, The Affiliated Hospital of Beijing Institute of Technology, Advanced Technology Research Institute, School of Life Science, Beijing Institute of Technology, Beijing, China
2. State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing, China
3. Advanced Technology Research Institute, Beijing Institute of Technology, Jinan, China
4. Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, State Key Laboratory of Digital Medical Engineering, Jiangsu Provincial Key Laboratory of Critical Care Medicine, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Southeast University, Nanjing, China
5. Co-Innovation Center of Neuroregeneration, Nantong University, Nantong, China
6. Department of Otolaryngology Head and Neck Surgery, Sichuan Provincial People’s Hospital, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China
7. Southeast University Shenzhen Research Institute, Shenzhen, China
Corresponding author:
qijieyu@bit.edu.cn
renjiec@seu.edu.cn
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History+
Received
Accepted
Published Online
2025-11-21
2026-04-29
2026-08-26
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Abstract
Hearing loss is among the most common sensory impairments globally and remains a significant unmet clinical challenge. Current rehabilitative approaches, such as hearing aids and cochlear implants, provide partial functional compensation but fail to restore the structural and physiological integrity of the cochlea. This review summarizes progress in therapeutic development targeting key deafness-associated genes, including OTOF, GJB2, and SLC26A4, highlighting the rationale, current clinical evidence, and major translational challenges. We further discuss the potential of combining gene therapy with stem cell-based approaches to promote inner ear regeneration and restore sensorineural function. Emerging technologies such as CRISPR/Cas, adeno-associated virus, and induced pluripotent stem cell-derived otic progenitors have opened new avenues for causal correction and tissue reconstruction. Integrated therapeutic models that combine gene or stem cell therapy with cochlear implants are reshaping the treatment paradigm from symptom management to biological repair. Advances in gene editing, gene delivery, and stem cell technologies are establishing these approaches as promising treatments for hereditary hearing loss, with ongoing progress expected to accelerate clinical translation despite remaining challenges in delivery efficiency, immune responses, ethics, and long-term efficacy. Collectively, these advances mark a pivotal step toward personalized and regenerative interventions for hereditary hearing loss.
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