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COMPOSITIONS AND METHODS OF SUPPRESSING EXPRESSION OF FACTOR VII GENES

机译:抑制因子VII基因表达的组合物和方法

摘要

1. A double-stranded ribonucleic acid molecule capable of suppressing expression of the factor VII gene in vitro by at least 70%. 2. The double-stranded ribonucleic acid molecule according to claim 1, wherein said double-stranded ribonucleic acid molecule comprises a sense and antisense strand, and an antisense strand is at least partially complementary to the sense strand, the sense strand comprising a sequence that is at least 90% identical to the part of the mRNA encoding factor VII, wherein said sequence (i) is localized in the complementarity region of said sense chain to said antisense chain, and (ii) said sequence with Toit of at least 30 nucleotides in dlinu.3. A double-stranded ribonucleic acid molecule according to one of claims 1 or 2, comprising nucleotides 1-19 of the sequences SEQ ID No: 413, 414, 415, 416, 417, 418, 419, 420, 421, 422, 423, 424, 425 , 426, 427, 428, 429, 430, 431, 432, 433, 434, 435, 436, 437, and 438.4. The double-stranded ribonucleic acid molecule according to claim 3, wherein the antisense strand further comprises a protrusion of 1-5 nucleotides from the 3 ′ end, preferably 1-2 nucleotides in length. The double-stranded ribonucleic acid molecule according to claim 4, wherein the protrusion of the antisense strand includes uracil or nucleotides that are at least 90% complementary to the mRNA encoding factor VII.6. The double-stranded ribonucleic acid molecule according to claim 3, wherein the sense strand further comprises a protrusion of 1-5 nucleotides, preferably 1-2 1-2 nucleotides, from the 3'-end. The double-stranded ribonucleic acid molecule according to claim 3, wherein the protrusion of the sense strand includes uracil or nucleotides that are at least 90
机译:1.一种双链核糖核酸分子,其能够在体外将因子VII基因的表达抑制至少70%。 2.根据权利要求1所述的双链核糖核酸分子,其中所述双链核糖核酸分子包含有义和反义链,并且反义链与所述有义链至少部分互补,所述有义链包含如下序列:与编码因子VII的mRNA的部分至少90%相同,其中所述序列(i)位于所述有义链与所述反义链的互补性区域,和(ii)具有至少30个核苷酸的Toit的所述序列在dlinu.3。 3.根据权利要求1或2之一的双链核糖核酸分子,其包含序列SEQ ID No:413、414、415、416、417、418、419、420、421、422、423的核苷酸1-19, 424、425、426、427、428、429、430、431、432、433、434、435、436、437和438.4。 4.根据权利要求3所述的双链核糖核酸分子,其中,所述反义链还包括从3'端开始的1-5个核苷酸的突出,优选长度为1-2个核苷酸的突出。 5.根据权利要求4所述的双链核糖核酸分子,其中,所述反义链的突出部包含尿嘧啶或与编码VII.6的mRNA至少90%互补的核苷酸。 4.根据权利要求3所述的双链核糖核酸分子,其中,所述有义链还包含从3'末端突出1-5个核苷酸,优选1-2个1-2个核苷酸的突出部。 4.根据权利要求3所述的双链核糖核酸分子,其中,所述有义链的突出部包括尿嘧啶或至少90个核苷酸。

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