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Lipophilic derivatives of double-stranded ribonucleic acid
专利权人:
Alnylam Europe AG
发明人:
John, Matthias,Limmer, Stefan,Lorenz, Christina,Hadwiger, Philipp,Vornlocher, Hans-Peter
申请号:
AU2008203538
公开号:
AU2008203538B2
申请日:
2008.08.07
申请国别(地区):
AU
年份:
2012
代理人:
摘要:
#$%^&*AU2008203538B220120517.pdf#####ABSTRACT 5 The present invention relates to a double-stranded ribonucleic acid (dsRNA) having improved efficiency of inhibition of gene expression, methods of making the dsRNA, and pharmaceutical compositions comprising the dsRNA. The dsRNA comprises an RNA strand (complementary 10 RNA strand) having a region which is complementary to an RNA transcript of at least a part of a target gene, and at least one covalently linked lipophilic group. The dsRNA are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of 1 the target gene. The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene. N: Sydney Cases Patent57000-57999 P57840.AU.1 Specis P57840.AU.1 Specification 2008-8-5.doc 5108/081 /5 NaHCO3 CH2 I2 0 00C ->; RT L 0 N (8 -9: 93%) 9 f~CCb ;N-K DIPEA0 (9 - 10: 70,4%/) 1 Fig. 1
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