自然杂志 ›› 2021, Vol. 43 ›› Issue (1): 39-44.doi: 10.3969/j.issn.0253-9608.2021.01.006

• 专题综述 • 上一篇    下一篇

AAV介导的听力损失基因治疗进展 

柯君子①②,谈方志①②,钟桂生①②   

  1. ①上海科技大学 iHuman研究所,上海 201210;② 上海科技大学 生命科学与技术学院,上海 201210 
  • 收稿日期:2020-10-01 出版日期:2021-02-25 发布日期:2021-02-25
  • 通讯作者: 柯君子,通信作者,研究方向:耳聋基因治疗。 谈方志,通信作者,研究方向:耳聋基因治疗。 钟桂生,通信作者,国家青年千人计划入选者,研究方向:耳聋基因治疗。

Advances in AAV-mediated gene therapy for hearing loss 

KE Junzi①②, TAN Fangzhi①②, ZHONG Guisheng①②   

  1. ①iHuman Institute, ShanghaiTech University, Shanghai 201210, China; ② School of Life Science and Technology, ShanghaiTech University, Shanghai 201210, China
  • Received:2020-10-01 Online:2021-02-25 Published:2021-02-25

摘要: 听力损失是常见的感觉障碍之一,尚没有非设备手段可以恢复听力。近年来听力疾病的基因疗法正在走向科研研究前端并逐步迈向临床。腺相关病毒(AAV)是目前最常用的基因治疗载体,在听力疾病的基因治疗中发挥重要作用。文章简 要综述了天然AAV的结构与分型,以Anc80L65、AAV2.7m8、AAV-ie为例阐述AAV介导的听力损失基因治疗的可能性, 并展望其未来的进展。

关键词:  , AAV;听力损失;基因治疗

Abstract: Hearing loss is one of the common sensory disorders, and there is no non-device means to restore hearing. In recent years, gene therapy for hearing diseases has gradually moved to the forefront of scientific research and clinical practice. Adeno-associated virus(AAV)is the most commonly used gene therapy vector and plays an important role in the gene therapy of hearing diseases. In this paper, the structure and type of natural AAV were briefly reviewed, and the possibility of AAV-mediated gene therapy for hearing loss was discussed with Anc80L65, AAV2.7m8 and AAV-ie as examples, and the future progress of AAV-mediated gene therapy for hearing loss was also discussed.