Current AAV-mediated gene therapy in sensorineural hearing loss  

作　　者：Jieyu Qi Xiaolong Fu Liyan Zhang Fangzhi Tan Nianci Li Qiuhan Sun Xiaojie Hu Zuhong He Ming Xia Renjie Chai 

机构地区：[1]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 [2]Department of Neurology,Aerospace Center Hospital,School of Life Science,Beijing Institute of Technology,Beijing 100081,China [3]State Key Laboratory of Hearing and Balance Science,Beijing Institute of Technology,Beijing 100081,China [4]Advanced Technology Research Institute,Beijing Institute of Technology,Jinan 250300,China [5]Department of Otorhinolaryngology-Head and Neck Surgery,Zhongnan Hospital of Wuhan University,Wuhan 430071,China [6]Department of Otolaryngology,Shandong Provincial Hospital Affiliated to Shandong First Medical University,Jinan 250021,China [7]Medical Science and Technology Innovation Center,Shandong First Medical University&Shandong Academy of Medical Sciences,Jinan 250021,China [8]Co-Innovation Center of Neuroregeneration,Nantong University,Nantong 226001,China [9]Department of Otolaryngology Head and Neck Surgery,Sichuan Provincial People's Hospital,University of Electronic Science and Technology of China,Chengdu 610072,China [10]Southeast University Shenzhen Research Institute,Shenzhen 518063,China

出　　处：《Fundamental Research》2025年第1期192-202,共11页自然科学基础研究(英文版)

基　　金：supported by the National Key Research and Development Program of China(2020YFA0113600,2021YFA1101300,2020YFA0112503);Strategic Priority Research Program of the Chinese Academy of Science(XDA16010303);the National Natural Science Foundation of China(82000984,82030029,81970882,81900937 and 82071013);Natural Science Foundation from Jiangsu Province(BE2019711);the China National Postdoctoral Program for Innovative Talents(BX20200082);the China Postdoctoral Science Foundation(2020M681468);the Science and Technology Department of Sichuan Province(2021YFS0371);Shenzhen Fundamental Research Program(JCYJ20190814093401920,JCYJ20210324125608022);Open Research Fund of State Key Laboratory of Genetic Engineering,Fudan University(SKLGE-2109);the Fundamental Research Funds for the Central Universities.

摘　　要：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.

关 键 词：Hearing loss COCHLEA Gene therapy Adeno-associated virus Auditory repair 

分 类 号：R764[医药卫生—耳鼻咽喉科]