Recent development of AAV-based gene therapies for inner ear disorders

doi:10.1038/s41434-020-0155-7

	Recent development of AAV-based gene therapies for inner ear disorders
	Lan, Yiyang1,2 ; Tao, Yong 3; Wang, Yunfeng 4,5; Ke, Junzi1,2 ; Yang, Qiuxiang1,2 ; Liu, Xiaoyi1,2 ; Su, Bing1,2 ; Wu, Yiling2 ; Lin, Chao-Po2 ; Zhong, Guisheng1,2
	2020-08
发表期刊	GENE THERAPY
ISSN	0969-7128
EISSN	1476-5462
发表状态	已发表
DOI	10.1038/s41434-020-0155-7
摘要	Gene therapy for auditory diseases is gradually maturing. Recent progress in gene therapy treatments for genetic and acquired hearing loss has demonstrated the feasibility in animal models. However, a number of hurdles, such as lack of safe viral vector with high efficiency and specificity, robust deafness large animal models, translating animal studies to clinic etc., still remain to be solved. It is necessary to overcome these challenges in order to effectively recover auditory function in human patients. Here, we review the progress made in our group, especially our efforts to make more effective and cell type-specific viral vectors for targeting cochlea cells.
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收录类别	SCI ; SCIE
语种	英语
资助项目	National Natural Science Foundation of China[81970878][31771130]
WOS研究方向	Biochemistry & Molecular Biology ; Biotechnology & Applied Microbiology ; Genetics & Heredity ; Research & Experimental Medicine
WOS类目	Biochemistry & Molecular Biology ; Biotechnology & Applied Microbiology ; Genetics & Heredity ; Medicine, Research & Experimental
WOS记录号	WOS:000533843300001
出版者	NATURE PUBLISHING GROUP
WOS关键词	ADENOASSOCIATED VIRUS VECTORS ; MOUSE MODEL ; HAIR-CELLS ; SUPPORTING CELLS ; PROTEIN EXPRESSION ; HEARING-LOSS ; IN-VITRO ; MAMMALIAN COCHLEA ; TRANSDUCTION ; MUTATIONS
原始文献类型	Review ; Early Access
引用统计
文献类型	期刊论文
条目标识符	https://kms.shanghaitech.edu.cn/handle/2MSLDSTB/121134
专题	生命科学与技术学院_硕士生生命科学与技术学院_PI研究组_林照博组 iHuman研究所_PI研究组_钟桂生组生命科学与技术学院_本科生
通讯作者	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.Shanghai Jiao Tong Univ, Shanghai Peoples Hosp 9, Dept Otolaryngol Head & Neck Surg, Sch Med, Shanghai 200011, Peoples R China 4.Fudan Univ, ENT Inst, Shanghai 200031, Peoples R China 5.Fudan Univ, Otorhinolaryngol Dept, Eye & ENT Hosp, NHC Key Lab Hearing Med, Shanghai 200031, Peoples R China
第一作者单位	iHuman研究所; 生命科学与技术学院
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	Lan, Yiyang,Tao, Yong,Wang, Yunfeng,et al. Recent development of AAV-based gene therapies for inner ear disorders[J]. GENE THERAPY,2020.
APA	Lan, Yiyang.,Tao, Yong.,Wang, Yunfeng.,Ke, Junzi.,Yang, Qiuxiang.,...&Zhong, Guisheng.(2020).Recent development of AAV-based gene therapies for inner ear disorders.GENE THERAPY.
MLA	Lan, Yiyang,et al."Recent development of AAV-based gene therapies for inner ear disorders".GENE THERAPY (2020).