Current AAV-mediated gene therapy in sensorineural hearing loss

The number of patients with hearing loss is on the rise due to congenital abnormalities, degenerative changes in old age, and acquired injuries such as virus or ototoxic drug-induced diseases. Hearing loss is a refractory and disabling disease that has serious negative effects on quality of life. Th...

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Main Authors: Jieyu Qi, Xiaolong Fu, Liyan Zhang, Fangzhi Tan, Nianci Li, Qiuhan Sun, Xiaojie Hu, Zuhong He, Ming Xia, Renjie Chai
Format: Article
Language:English
Published: KeAi Communications Co. Ltd. 2025-01-01
Series:Fundamental Research
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Online Access:http://www.sciencedirect.com/science/article/pii/S2667325822003569
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author Jieyu Qi
Xiaolong Fu
Liyan Zhang
Fangzhi Tan
Nianci Li
Qiuhan Sun
Xiaojie Hu
Zuhong He
Ming Xia
Renjie Chai
author_facet Jieyu Qi
Xiaolong Fu
Liyan Zhang
Fangzhi Tan
Nianci Li
Qiuhan Sun
Xiaojie Hu
Zuhong He
Ming Xia
Renjie Chai
author_sort Jieyu Qi
collection DOAJ
description The number of patients with hearing loss is on the rise due to congenital abnormalities, degenerative changes in old age, and acquired injuries such as virus or ototoxic drug-induced diseases. Hearing loss is a refractory and disabling disease that has serious negative effects on quality of life. The pathology of hearing loss in the inner ear is characterized by varying degrees of damage to the cochlear sensory epithelium cells (such as hair cells and supporting cells), stria vascularis (including marginal, intermediate and basal cells) and spiral ganglion neurons. Regeneration or direct repair of damaged cells in the inner ear is an effective way to treat sensorineural deafness. It is currently possible to regenerate hair cells to treat sensorineural hearing loss by FX-322, a small molecule drug in clinical trials. With the development of genetic engineering technology, gene therapy has brought a promising treatment strategy for many previously intractable diseases. Gene therapy has been regarded as a promising method in the treatment and rehabilitation of sensorineural hearing loss, and recombinant adeno-associated virus gene therapy has been widely used in fundamental research into hearing loss treatments. At present, gene therapy for hearing loss is transitioning from feasibility studies to explorations of its safety and its therapeutic potential. The present article reviews the concepts, strategies, and applications of gene therapy mediated by recombinant adeno-associated viruses in the field of hearing loss treatment.
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spelling doaj-art-4eb91d1db26d467693e22f20d6618ece2025-01-29T05:02:27ZengKeAi Communications Co. Ltd.Fundamental Research2667-32582025-01-0151192202Current AAV-mediated gene therapy in sensorineural hearing lossJieyu Qi0Xiaolong Fu1Liyan Zhang2Fangzhi Tan3Nianci Li4Qiuhan Sun5Xiaojie Hu6Zuhong He7Ming Xia8Renjie Chai9State 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; Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing 100081, China; State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing 100081, China; Advanced Technology Research Institute, Beijing Institute of Technology, Jinan 250300, 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, ChinaDepartment of Otorhinolaryngology-Head and Neck Surgery, Zhongnan Hospital of Wuhan University, Wuhan 430071, China; Corresponding authors.Department of Otolaryngology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan 250021, China; Medical Science and Technology Innovation Center, Shandong First Medical University & Shandong Academy of Medical Sciences, Jinan 250021, China; Corresponding authors.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; Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing 100081, China; Co-Innovation Center of Neuroregeneration, Nantong University, Nantong 226001, China; Department of Otolaryngology Head and Neck Surgery, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu 610072, China; Southeast University Shenzhen Research Institute, Shenzhen 518063, China; Corresponding authors.The number of patients with hearing loss is on the rise due to congenital abnormalities, degenerative changes in old age, and acquired injuries such as virus or ototoxic drug-induced diseases. Hearing loss is a refractory and disabling disease that has serious negative effects on quality of life. The pathology of hearing loss in the inner ear is characterized by varying degrees of damage to the cochlear sensory epithelium cells (such as hair cells and supporting cells), stria vascularis (including marginal, intermediate and basal cells) and spiral ganglion neurons. Regeneration or direct repair of damaged cells in the inner ear is an effective way to treat sensorineural deafness. It is currently possible to regenerate hair cells to treat sensorineural hearing loss by FX-322, a small molecule drug in clinical trials. With the development of genetic engineering technology, gene therapy has brought a promising treatment strategy for many previously intractable diseases. Gene therapy has been regarded as a promising method in the treatment and rehabilitation of sensorineural hearing loss, and recombinant adeno-associated virus gene therapy has been widely used in fundamental research into hearing loss treatments. At present, gene therapy for hearing loss is transitioning from feasibility studies to explorations of its safety and its therapeutic potential. The present article reviews the concepts, strategies, and applications of gene therapy mediated by recombinant adeno-associated viruses in the field of hearing loss treatment.http://www.sciencedirect.com/science/article/pii/S2667325822003569Hearing lossCochleaGene therapyAdeno-associated virusAuditory repair
spellingShingle Jieyu Qi
Xiaolong Fu
Liyan Zhang
Fangzhi Tan
Nianci Li
Qiuhan Sun
Xiaojie Hu
Zuhong He
Ming Xia
Renjie Chai
Current AAV-mediated gene therapy in sensorineural hearing loss
Fundamental Research
Hearing loss
Cochlea
Gene therapy
Adeno-associated virus
Auditory repair
title Current AAV-mediated gene therapy in sensorineural hearing loss
title_full Current AAV-mediated gene therapy in sensorineural hearing loss
title_fullStr Current AAV-mediated gene therapy in sensorineural hearing loss
title_full_unstemmed Current AAV-mediated gene therapy in sensorineural hearing loss
title_short Current AAV-mediated gene therapy in sensorineural hearing loss
title_sort current aav mediated gene therapy in sensorineural hearing loss
topic Hearing loss
Cochlea
Gene therapy
Adeno-associated virus
Auditory repair
url http://www.sciencedirect.com/science/article/pii/S2667325822003569
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AT niancili currentaavmediatedgenetherapyinsensorineuralhearingloss
AT qiuhansun currentaavmediatedgenetherapyinsensorineuralhearingloss
AT xiaojiehu currentaavmediatedgenetherapyinsensorineuralhearingloss
AT zuhonghe currentaavmediatedgenetherapyinsensorineuralhearingloss
AT mingxia currentaavmediatedgenetherapyinsensorineuralhearingloss
AT renjiechai currentaavmediatedgenetherapyinsensorineuralhearingloss