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

Fangzhi Tan (), Cenfeng Chu, Jieyu Qi, Wenyan Li, Dan You, Ke Li, Xin Chen, Weidong Zhao, Cheng Cheng, Xiaoyi Liu, Yunbo Qiao, Bing Su, Shuijin He, Chao Zhong, Huawei Li (), Renjie Chai () and Guisheng Zhong ()
Additional contact information
Fangzhi Tan: ShanghaiTech University
Cenfeng Chu: ShanghaiTech University
Jieyu Qi: Southeast University
Wenyan Li: Fudan University
Dan You: Fudan University
Ke Li: University of the Chinese Academy of Sciences
Xin Chen: ShanghaiTech University
Weidong Zhao: Fudan University
Cheng Cheng: Jiangsu Provincial Key Medical Discipline (Laboratory)
Xiaoyi Liu: ShanghaiTech University
Yunbo Qiao: Guangzhou University
Bing Su: ShanghaiTech University
Shuijin He: ShanghaiTech University
Chao Zhong: ShanghaiTech University
Huawei Li: Fudan University
Renjie Chai: Southeast University
Guisheng Zhong: ShanghaiTech University

Nature Communications, 2019, vol. 10, issue 1, 1-10

Abstract: Abstract 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.

Date: 2019
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DOI: 10.1038/s41467-019-11687-8

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