Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65

Chin Ju Hu*, Ying Chang Lu, Yi Hsiu Tsai, Haw Yuan Cheng, Hiroki Takeda, Chun Ying Huang, Ru Xiao, Chuan Jen Hsu, Jin Wu Tsai, Luk H. Vandenberghe, Chen Chi Wu, Yen Fu Cheng

*此作品的通信作者

研究成果: Article同行評審

10 引文 斯高帕斯(Scopus)

摘要

This study demonstrates that Anc80L65, a synthetic AAV vector validated to exhibit high expression in the inner ears of neonatal and adult mice, can target both cochlear and vestibular hair cells and spiral ganglion neurons when delivered in utero. These findings support the potential use of Anc80L65 in prenatal gene therapy of hereditary hearing loss.

原文English
頁(從 - 到)493-500
頁數8
期刊Molecular Therapy - Methods and Clinical Development
18
DOIs
出版狀態Published - 11 9月 2020

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