Details

Autor(en) / Beteiligte

• 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

Titel

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

Ist Teil von

• Nature communications, 2019-08, Vol.10 (1), p.3733-10, Article 3733

Ort / Verlag

England: Nature Publishing Group

Erscheinungsjahr

2019

Quelle

MEDLINE

Beschreibungen/Notizen

• 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.