{"id":"doi:10.1038/s41467-019-11687-8","kind":"article","title":"AAV-ie enables safe and efficient gene transfer to inner ear cells","authors":["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","Guisheng Zhong"],"author_count":17,"year":2019,"published":"2019-08-19","venue":"Nature Communications","cited_by":232,"doi":"10.1038/s41467-019-11687-8","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41467-019-11687-8","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41467-019-11687-8","updated":null,"pmid":"31427575","pmcid":null,"openalex_id":"W2969527757","pdf_url":"https://www.nature.com/articles/s41467-019-11687-8.pdf","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.","abstract_license":"CC-BY (publisher license, via OpenAlex)","license":"cc-by","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":["Virus-based gene therapy research"],"details":null,"provenance":"openalex","attribution":"Abstract of \"AAV-ie enables safe and efficient gene transfer to inner ear cells\" by Fangzhi Tan, Cenfeng Chu, Jieyu Qi et al., https://doi.org/10.1038/s41467-019-11687-8, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"gene-cell-therapy","name":"Gene & Cell Therapy","domain_slug":"medicine"}],"cited_in_dossiers":[]}