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GENE THERAPY FOR AUTOSOMAL DOMINANT DISEASES
专利权人:
发明人:
申请号:
EP16787314.0
公开号:
EP3289080A2
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.
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中国工程科技知识中心
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