AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives
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 diso...
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| Language: | English |
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Wiley
2024-12-01
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| Series: | Advanced Science |
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| Online Access: | https://doi.org/10.1002/advs.202402166 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Liyan Zhang |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-476f4beb1f424169ab832ba9b5fd883d |
| institution | Kabale University |
| issn | 2198-3844 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Wiley |
| record_format | Article |
| series | Advanced Science |
| spelling | doaj-art-476f4beb1f424169ab832ba9b5fd883d2024-12-18T14:18:10ZengWileyAdvanced Science2198-38442024-12-011147n/an/a10.1002/advs.202402166AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and PerspectivesLiyan Zhang0Fangzhi Tan1Jieyu Qi2Yicheng Lu3Xiaohan Wang4Xuehan Yang5Xiangyan Chen6Xinru Zhang7Jinyi Fan8Yinyi Zhou9Li Peng10Nianci Li11Lei Xu12Shiming Yang13Renjie Chai14State 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaState 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 ChinaOtovia Therapeutics Inc. Suzhou 215101 ChinaState 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 ChinaDepartment of Otolaryngology‐Head and Neck Surgery Shandong Provincial ENT Hospital Shandong University Jinan Shandong 250022 ChinaSenior Department of Otolaryngology Head and Neck Surgery Chinese PLA General Hospital Chinese PLA Medical School Beijing 100853 ChinaState 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 ChinaAbstract 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.https://doi.org/10.1002/advs.202402166AAVgene therapyhereditary deafness |
| spellingShingle | 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 AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives Advanced Science AAV gene therapy hereditary deafness |
| title | AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives |
| title_full | AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives |
| title_fullStr | AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives |
| title_full_unstemmed | AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives |
| title_short | AAV‐mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives |
| title_sort | aav mediated gene therapy for hereditary deafness progress and perspectives |
| topic | AAV gene therapy hereditary deafness |
| url | https://doi.org/10.1002/advs.202402166 |
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