The Trustees of Columbia University in the City of New York
发明人:
WU, Wen-hsuan,TSAI, Yi-ting,CHAN, Lawrence,TSANG, Stephen, H.
申请号：
EP16787314
公开号：
EP3289080A4
申请日:
2016.05.02
申请国别(地区):
EP
年份:
2018
代理人:
摘要：
The present disclosure provides methods for treating autosomal dominant diseases in a subject. In some aspects, the methods involve the use of a gene editing enzyme with a pair of unique guide RNA sequences that targets both mutant and wildtype forms of autosomal dominant disease-related gene for destruction in cells, and then supplying the cells with wildtype autosomal dominant disease-related gene cDNA which is codon modified to evade recognition by the guide RNAs. These methods are broadly applicable to any autosomal dominant disease.
