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Compositions and methods for inhibiting expression of huntingtin gene
专利权人:
发明人:
申请号:
EP11000550.1
公开号:
EP2325315B1
申请日:
2006.10.27
申请国别(地区):
EP
年份:
2014
代理人:
摘要:
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition and methods for inhibiting the expression of the huntingtin gene in a cell.
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