AAV-ie-K558R mediated cochlear gene therapy and hair cell regeneration  

作　　者：Yong Tao Xiaoyi Liu Liu Yang Cenfeng Chu Fangzhi Tan Zehua Yu Junzi Ke Xiang Li Xiaofei Zheng Xingle Zhao Jieyu Qi Chao-Po Lin Renjie Chai Guisheng Zhong Hao Wu 

机构地区：[1]Department of Otolaryngology-Head and Neck Surgery,Shanghai Ninth People’s Hospital,Shanghai Jiao Tong University School of Medicine,Shanghai 200011,PR China [2]Ear Institute,Shanghai Jiao Tong University School of Medicine,Shanghai 200011,PR China [3]Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases,Shanghai 200011,PR China [4]iHuman Institute,ShanghaiTech University,Shanghai 201210,PR China [5]School of Life Science and Technology,ShanghaiTech University,Shanghai 201210,PR China [6]State Key Laboratory of Bioelectronics,Department of Otolaryngology Head and Neck Surgery,Zhongda Hospital,School of Life Sciences and Technology,Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research,Southeast University,Nanjing 210096,PR China [7]Co-Innovation Center of Neuroregeneration,Nantong University,Nantong 226001,PR China [8]Institute for Stem Cell and Regeneration,Chinese Academy of Science,Beijing,PR China [9]Beijing Key Laboratory of Neural Regeneration and Repair,Capital Medical University,100069 Beijing,PR China

出　　处：《Signal Transduction and Targeted Therapy》2022年第5期1764-1772,共9页信号转导与靶向治疗（英文）

基　　金：We thank the Shanghai Municipal Government and ShanghaiTech University,and the Bioimaging Core Facilities of the iHuman Institute and the animal facility of National Center for Protein Science Shanghai for their financial support.Hao Wu was supported by the Key Project of the National Natural Science Foundation of China(NSFC 81730028);the National Key Technology Research and Development Program of the Ministry of Science and Technology of China(2017YFC1001800);Shanghai Key Laboratory of Translational Medicine on Ear and Nose diseases(14DZ2260300);Shanghai Municipal Science and Technology Major Project(2018SHZDZX05);the Innovative Research Team of High-level Local Universities in Shanghai.This work was supported by the National Natural Science Foundation of China(31771130(G.Z.);the 2015 Thousand Youth Talents Plan of China(G.Z.,).This work was supported by the National Natural Science Foundation of China(82122019,Y.T.);the program for professors with special appointments(Eastern Scholar,Y.T.)at Shanghai Institutions of Higher Learning.

摘　　要：The cochlea consists of multiple types of cells,including hair cells,supporting cells and spiral ganglion neurons,and is responsible for converting mechanical forces into electric signals that enable hearing.Genetic and environmental factors can result in dysfunctions of cochlear and auditory systems.In recent years,gene therapy has emerged as a promising treatment in animal deafness models.One major challenge of the gene therapy for deafness is to effectively deliver genes to specific cells of cochleae.

关 键 词：COCHLEAR AUDITORY DEAFNESS 

分 类 号：R764[医药卫生—耳鼻咽喉科]