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发明公开
CN104471062A 抑制KRAS基因表达的RNAi医药组合物 无效 - 撤回

抑制KRAS基因表达的RNAi医药组合物
摘要：
本发明提供一种抑制KRAS基因表达的组合物等，其含有脂质粒子，所述脂质粒子含有作为药物的双链核酸和式(I)表示的阳离子性脂质，所述双链核酸中，反义链具有与序列号1～3中任一的KRAS基因的mRNA的连续至少19个碱基的序列互补的碱基序列；式(I)中，R1及R2相同或不同，为碳原子数为12～24的直链状或支链状的烷基、链烯基或炔基，L1及L2相同或不同，为-CO-O-或-O-CO-，a及b相同或不同，为1～3，R3为氢原子、碳原子数为1～6的烷基或碳原子数为3～6的链烯基。
摘要（英）：

Provided are: a composition which can suppress the expression of KRAS gene and comprises a double-stranded nucleic acid that serves as a medicinal agent and lipid particles, wherein an antisense strand in the double-stranded nucleic acid comprises a nucleotide sequence complementary to a sequence that is represented by any one of SEQ ID NOs: 1 to 3 and is composed of at least 19 contiguous nucleotides contained in mRNA for KRAS gene, and each of the lipid particles comprises a cationic lipid represented by formula (I) (wherein R1 and R2 may be the same as or different from each and independently represent a linear or branched alkyl, alkenyl or alkynyl group having 12 to 24 carbon atoms, L1 and L2 may be the same as or different from each other and independently represent -CO-O- or -O-CO-, a and b may be the same as or different from each and independently represent 1 to 3, and R3 represents a hydrogen atom, an alkyl group having 1 to 6 carbon atoms or an alkenyl group having 3 to 6 carbon atoms); and others.

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