AAV-ie enables safe and efficient gene transfer to inner ear cells
文献类型：期刊论文
| 作者 | Tan, Fangzhi; Chu, Cenfeng; Qi, Jieyu; Li, Wenyan; You, Dan; Li, Ke; Chen, Xin; Zhao, Weidong; Cheng, Cheng; Liu, Xiaoyi |
| 刊名 | NATURE COMMUNICATIONS
 |
| 出版日期 | 2019-08-19 |
| 卷号 | 10 |
| ISSN号 | 2041-1723 |
| DOI | 10.1038/s41467-019-11687-8 |
| 文献子类 | Article |
| 英文摘要 | 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. |
| WOS研究方向 | Science & Technology - Other Topics |
| 语种 | 英语 |
| 出版者 | NATURE PUBLISHING GROUP |
| 源URL | [http://ir.sic.ac.cn/handle/331005/26901]  |
| 专题 | 中国科学院上海硅酸盐研究所 |
| 推荐引用方式 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). |
入库方式： OAI收割
来源：上海硅酸盐研究所
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