Hinrich Staecker,Xue Zhong Liu,Zheng-Yi Chen,Caesar James Ayala
申请号:
US16488103
公开号:
US20200248203A1
申请日:
2018.03.16
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Disclosed are compositions and methods that useful in the treatment and/or prevention of hearing loss caused by genetic mutation of the TMPRSS3 gene or the LOXHD1 gene. The compositions and methods disclosed herein use adeno-associated viral (AAV) vector gene delivery of TRMPSS3 or LOXHD1 into the inner ear to restore activity of the TMPRSS3 gene or the LOXHD1 gene, respectively, promote hair cell survival and restore hearing in patients suffering from hearing loss.