Melvin Maurice EVERS,Pavlina Stefanova KONSTANTINOVA,Raygene Michael MARTIER
申请号：
US16684028
公开号：
US20200199625A1
申请日:
2019.11.14
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The current invention relates to gene therapy approaches for the treatment of SCA3, in particular RNAi based gene therapy approaches utilizing a total knockdown approach. The inventors provide for selected target regions and/or target sequences for which highly efficient knockdown of the ATXN3 gene expression can be advantageously obtained in human neuronal cells and in mouse models relevant for SCA3.
