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

Author

Listed:
  • Fangzhi Tan

    (ShanghaiTech University)

  • Cenfeng Chu

    (ShanghaiTech University
    ShanghaiTech University
    University of the Chinese Academy of Sciences)

  • Jieyu Qi

    (Southeast University
    Nantong University)

  • Wenyan Li

    (Fudan University)

  • Dan You

    (Fudan University)

  • Ke Li

    (University of the Chinese Academy of Sciences
    ShanghaiTech University
    Chinese Academy of Sciences)

  • Xin Chen

    (ShanghaiTech University
    University of the Chinese Academy of Sciences)

  • Weidong Zhao

    (Fudan University)

  • Cheng Cheng

    (Jiangsu Provincial Key Medical Discipline (Laboratory)
    Research Institute of Otolaryngology)

  • Xiaoyi Liu

    (ShanghaiTech University
    ShanghaiTech University
    University of the Chinese Academy of Sciences)

  • Yunbo Qiao

    (Guangzhou University)

  • Bing Su

    (ShanghaiTech University
    ShanghaiTech University
    University of the Chinese Academy of Sciences)

  • Shuijin He

    (ShanghaiTech University)

  • Chao Zhong

    (ShanghaiTech University)

  • Huawei Li

    (Fudan University)

  • Renjie Chai

    (Southeast University
    Nantong University
    Fudan University
    Chinese Academy of Science)

  • Guisheng Zhong

    (ShanghaiTech University
    ShanghaiTech University
    Nantong University)

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.

Suggested Citation

  • 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 & Gui, 2019. "AAV-ie enables safe and efficient gene transfer to inner ear cells," Nature Communications, Nature, vol. 10(1), pages 1-10, December.
    • Handle: RePEc:nat:natcom:v:10:y:2019:i:1:d:10.1038_s41467-019-11687-8
    DOI: 10.1038/s41467-019-11687-8
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    Cited by:

    1. Xinhong Chen & Damien A. Wolfe & Dhanesh Sivadasan Bindu & Mengying Zhang & Naz Taskin & David Goertsen & Timothy F. Shay & Erin E. Sullivan & Sheng-Fu Huang & Sripriya Ravindra Kumar & Cynthia M. Aro, 2023. "Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates," Nature Communications, Nature, vol. 14(1), pages 1-19, December.

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