发明名称 |
Lipid-modified double-stranded RNA having potent RNA interference effect |
摘要 |
An object of the present invention is to provide a novel double-stranded RNA that has high nuclease resistance and high cellular uptake efficiency, and that is capable of producing an excellent RNA interference effect. The present invention provides a lipid-modified double-stranded RNA comprising a sense strand having a nucleotide sequence complementary to a target sequence, and an antisense strand having a nucleotide sequence complementary to the sense strand, the double-stranded RNA being capable of inhibiting the expression of the target gene, the sense strand having a lipid linked to at least one of the first to sixth nucleotides from the 5′ end side directly or via a linker. |
申请公布号 |
US8981074(B2) |
申请公布日期 |
2015.03.17 |
申请号 |
US200812738797 |
申请日期 |
2008.10.24 |
申请人 |
National Institute of Advanced Industrial Science and Technology;Otsuka Pharmaceutical Co., Ltd. |
发明人 |
Kubo Takanori;Ohba Hideki;Toyobuku Hidekazu;Hayashi Hirotake |
分类号 |
C07H21/04;A61K47/48 |
主分类号 |
C07H21/04 |
代理机构 |
Sughrue Mion, PLLC |
代理人 |
Sughrue Mion, PLLC |
主权项 |
1. A lipid-modified double-stranded RNA comprising:
an antisense strand having a nucleotide sequence complementary to a target sequence in a target gene, and a sense strand having a nucleotide sequence complementary to the antisense strand, the double-stranded RNA being blunt-ended on the 5′ end side of the sense strand, and being blunt-ended or having a dangling end on the 3′ end side of the sense strand, or alternatively the double-stranded RNA having dangling ends on both the 5′ and 3′ end sides of the sense strand, the sense strand having a lipid linked to at least one of the first to sixth nucleotides from the 5′ end via a linker, the lipid being a fatty acid having 6 to 50 carbon atoms, andthe linker being represented by the structural formula: —NH—(CH2)n1— (L-4), wherein n1 is an integer of 1 to 40. |
地址 |
Tokyo JP |