journal article·2024-12Open access

AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives

Liyan Zhang, Fangzhi Tan, Jieyu Qi, Yicheng Lu, Xiaohan Wang, Xuehan Yang, Xiangyan Chen, Xinru Zhang, Jinyi Fan, Yinyi Zhou, Li Peng, Nianci Li, Lei Xu, Shiming Yang, Renjie Chai

Advanced Science· Volume 11· Issue 47

Abstract

Abstract Hereditary deafness is the most prevalent sensory deficit disorder, with over 100 identified deafness‐related genes. Clinical treatment options are currently limited to external devices like hearing aids and cochlear implants. Gene therapy has shown promising results in various genetic disorders and has emerged as a potential treatment for hereditary deafness. It has successfully restored hearing function in >20 types of genetic deafness model mice and can almost completely cure patients with hereditary autosomal recessvie deafness 9 (DFNB9) caused by the OTOFERLIN ( OTOF ) mutation, thus serving as a translational paradigm for gene therapy for other forms of genetic deafness. However, due to the complexity of the inner ear structure, the diverse nature of deafness genes, and variations in transduction efficiency among different types of inner ear cells targeted by adeno‐associated virus (AAV), precision gene therapy approaches are required for different genetic forms of deafness. This review provides a comprehensive overview of gene therapy for hereditary deafness, including preclinical studies and recent research advancements in this field as well as challenges associated with AAV‐mediated gene therapy.
Source: crossref · Quality: baseline

Research context

OpenAlex topics: Hearing, Cochlea, Tinnitus, Genetics · RNA regulation and disease · CRISPR and Genetic Engineering

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

State Key Laboratory of Digital Medical Engineering Department of Otolaryngology Head and Neck Surgery Zhongda Hospital School of Life Sciences and Technology School of Medicine Advanced Institute for Life and Health Jiangsu Province High‐Tech Key Laboratory for Bio‐Medical Research Southeast University Nanjing 210096 China

Co‐Innovation Center of Neuroregeneration Nantong University Nantong 226001 China

State Key Laboratory of Hearing and Balance Science Department of Neurology Aerospace Center Hospital School of Life Science Beijing Institute of Technology Beijing 100081 China

Otovia Therapeutics Inc. Suzhou 215101 China

Department of Otolaryngology‐Head and Neck Surgery Shandong Provincial ENT Hospital Shandong University Jinan Shandong 250022 China

Senior Department of Otolaryngology Head and Neck Surgery Chinese PLA General Hospital Chinese PLA Medical School Beijing 100853 China

State Key Laboratory of Hearing and Balance Science Beijing 100853 China

National Clinical Research Center for Otolaryngologic Diseases Beijing 100853 China

Key Laboratory of Hearing Science Ministry of Education Beijing 100853 China

Beijing Key Laboratory of Hearing Impairment Prevention and Treatment Beijing 100853 China

University of Electronic Science and Technology of China Chengdu 610072 China

Southeast University Shenzhen Research Institute Shenzhen 518063 China

Funding

National Key Research and Development Program of China · National Natural Science Foundation of China · Natural Science Foundation of Jiangsu Province · Science and Technology Department of Sichuan Province · Taishan Scholar Foundation of Shandong Province · China Postdoctoral Science Foundation

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