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AAV-ie enables safe and efficient gene transfer to inner ear cells
Tan, Fangzhi; Chu, Cenfeng; Qi, Jieyu; Li, Wenyan; You, Dan; Li, Ke; Chen, Xin; Zhao, Weidong; Cheng, Cheng; Liu, Xiaoyi; Qiao, Yunbo; Su, Bing; He, Shuijin; Zhong, Chao; Li, Huawei; Chai, Renjie; Zhong, Guisheng
2019-08-19
Source PublicationNATURE COMMUNICATIONS
ISSN2041-1723
Volume10
SubtypeArticle
AbstractHearing 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.
DOI10.1038/s41467-019-11687-8
Language英语
WOS Research AreaScience & Technology - Other Topics
PublisherNATURE PUBLISHING GROUP
Citation statistics
Document Type期刊论文
Identifierhttp://ir.sic.ac.cn/handle/331005/26901
Collection中国科学院上海硅酸盐研究所
Recommended Citation
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).
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