AAV-ie enables safe and efficient gene transfer to inner ear cells

doi:10.1038/s41467-019-11687-8

	AAV-ie enables safe and efficient gene transfer to inner ear cells
	Tan, Fangzhi1 ; Chu, Cenfeng1,2,3 ; Qi, Jieyu 4,5; Li, Wenyan 6,7; You, Dan 6,7; Li, Ke3,8,9 ; Chen, Xin2,3 ; Zhao, Weidong 6,7; Cheng, Cheng 10,11; Liu, Xiaoyi1,2,3 ; Qiao, Yunbo 12; Su, Bing1,2,3 ; He, Shuijin2 ; Zhong, Chao8 ; Li, Huawei 6,7; Chai, Renjie 4,5,6,7,13,14; Zhong, Guisheng1,2,5
	2019-08-19
发表期刊	NATURE COMMUNICATIONS (IF:14.7[JCR-2023],16.1[5-Year])
ISSN	2041-1723
卷号	10
发表状态	已发表
DOI	10.1038/s41467-019-11687-8
摘要	Hearing loss is the most common sensory disorder. While gene therapy has emerged as a promising treatment of inherited diseases like hearing loss, it is dependent on the identification of gene delivery vectors. Adeno-associated virus (AAV) vector-mediated gene therapy has been approved in the US for treating a rare inherited eye disease but no safe and efficient vectors have been identified that can target the diverse types of inner ear cells. Here, we identify an AAV variant, AAV-inner ear (AAV-ie), for gene delivery in mouse inner ear. Our results show that AAV-ie transduces the cochlear supporting cells (SCs) with high efficiency, representing a vast improvement over conventional AAV serotypes. Furthermore, after AAV-ie-mediated transfer of the Atoh1 gene, we find that many SCs trans-differentiated into new HCs. Our results suggest that AAV-ie is a useful tool for the cochlear gene therapy and for investigating the mechanism of HC regeneration.
收录类别	SCI ; SCIE
资助项目	National Natural Science Foundation of China[31771130] ; National Natural Science Foundation of China[31601185] ; National Natural Science Foundation of China[81622013] ; National Natural Science Foundation of China[81620108005]
WOS研究方向	Science & Technology - Other Topics
WOS类目	Multidisciplinary Sciences
WOS记录号	WOS:000481607000003
出版者	NATURE PUBLISHING GROUP
WOS关键词	SUPPORTING CELLS ; HAIR-CELLS ; MOUSE MODEL ; SCALA MEDIA ; HEARING ; THERAPY ; DELIVERY ; COCHLEA ; REPLACEMENT ; MATH1
原始文献类型	Article
引用统计	被引频次：153[WOS] [WOS记录] [WOS相关记录]
文献类型	期刊论文
条目标识符	https://kms.shanghaitech.edu.cn/handle/2MSLDSTB/64575
专题	生命科学与技术学院_本科生物质科学与技术学院_PI研究组_钟超组生命科学与技术学院_PI研究组_何水金组 iHuman研究所_PI研究组_钟桂生组生命科学与技术学院_硕士生生命科学与技术学院_博士生物质科学与技术学院_博士生
共同第一作者	Chu, Cenfeng; Qi, Jieyu; Li, Wenyan
通讯作者	Tan, Fangzhi; Li, Huawei; Chai, Renjie; Zhong, Guisheng
作者单位	1.ShanghaiTech Univ, iHuman Inst, Shanghai 201210, Peoples R China 2.ShanghaiTech Univ, Sch Life Sci & Technol, Shanghai 201210, Peoples R China 3.Univ Chinese Acad Sci, Beijing 100049, Peoples R China 4.Southeast Univ, MOE Key Lab Dev Genes & Human Dis, Inst Life Sci, Jiangsu Prov High Tech Key Lab Biomed Res, Nanjing 210096, Jiangsu, Peoples R China 5.Nantong Univ, Jiangsu Key Lab Neuroregenerat, Coinnovat Ctr Neuroregenerat, Nantong, Peoples R China 6.Fudan Univ, ENT Inst, Shanghai 200031, Peoples R China 7.Fudan Univ, Shanghai Engn Res Ctr Cochlear Implant,NHFPC, State Key Lab Med Neurobiol,Otorhinolaryngol Dept, Affiliated Eye & ENT Hosp,Key Lab Hearing Med, Shanghai 200031, Peoples R China 8.ShanghaiTech Univ, Sch Phys Sci & Technol, Shanghai 201210, Peoples R China 9.Chinese Acad Sci, Shanghai Inst Ceram, Shanghai 200050, Peoples R China 10.Nanjing Univ, Dept Otolaryngol Head & Neck Surg, Affiliated Drum Tower Hosp, Med Sch,Jiangsu Prov Key Med Discipline Lab, 321 Zhongshan Rd, Nanjing 210008, Jiangsu, Peoples R China 11.Res Inst Otolaryngol, 321 Zhongshan Rd, Nanjing 210008, Jiangsu, Peoples R China 12.Guangzhou Univ, Sch Life Sci, Precise Genome Engn Ctr, Guangzhou 510006, Guangdong, Peoples R China 13.Chinese Acad Sci, Inst Stem Cell & Regenerat, Beijing, Peoples R China 14.Capital Med Univ, Beijing Key Lab Neural Regenerat & Repair, Beijing 100069, Peoples R China
第一作者单位	iHuman研究所
通讯作者单位	iHuman研究所; 生命科学与技术学院
第一作者的第一单位	iHuman研究所
推荐引用方式 GB/T 7714	Tan, Fangzhi,Chu, Cenfeng,Qi, Jieyu,et al. AAV-ie enables safe and efficient gene transfer to inner ear cells[J]. NATURE COMMUNICATIONS,2019,10.
APA	Tan, Fangzhi.,Chu, Cenfeng.,Qi, Jieyu.,Li, Wenyan.,You, Dan.,...&Zhong, Guisheng.(2019).AAV-ie enables safe and efficient gene transfer to inner ear cells.NATURE COMMUNICATIONS,10.
MLA	Tan, Fangzhi,et al."AAV-ie enables safe and efficient gene transfer to inner ear cells".NATURE COMMUNICATIONS 10(2019).