Man Wang1  · Bei Wang1  · Minhao Yang8  · Fangzhi Tan1  · Jieyu Qi3,4,5  · Renjie Chai1,2,4,6,7

1 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 210096, China 

2 Co‑Innovation Center of Neuroregeneration, Nantong University, Nantong 226001, China 

3 Advanced Technology Research Institute, Beijing Institute of Technology, Jinan 250307, China 

4 Department of Radiology, School of Life Science, Zhuhai People’s Hospital, The Affiliated Hospital of Beijing Institute of Technology, Advanced Technology Research Institute, Beijing Institute of Technology, Beijing 100081, China 

5 State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing 100081, China 

6 Department of Otolaryngology Head and Neck Surgery, School of Medicine, Sichuan Provincial People’s Hospital, University of Electronic Science and Technology of China, Chengdu 610072, China 

7 Southeast University Shenzhen Research Institute, Shenzhen 518063, China 

8 School of Public Health, Nanjing Medical University, Nanjing 210029, China

Abstract

Hereditary deafness represents a significant global health challenge with limited therapeutic interventions. Most cases are caused by monogenic mutations inherited in an autosomal dominant or recessive manner, making them suitable targets for gene editing therapies. Recent advances in gene editing technologies have expanded the toolkit for precise genomic modification, including engineered nucleases for gene disruption, base editors (BEs) for point mutations, prime editors (PEs) for substitutions, insertions, and deletions, and mitochondrial editors for modifying mitochondrial DNA (mtDNA). These tools have demonstrated significant efficacy in mouse models of hereditary deafness, highlighting their clinical potential. However, given the high degree of genetic heterogeneity, gene editing technologies in this field remain in an early exploratory stage. In this review, we provide a comprehensive overview of the latest breakthroughs in gene-editing platforms and critically evaluate the potential benefits and existing hurdles to their clinical application for treating congenital hearing impairment.

Keywords

Gene editing technology; Hereditary hearing loss; Gene therapy; Hearing restoration

[SpringerLink]