БУМКРОТ Дэвид (US),БЕТТЕНКОРТ Брайан (US),ТУДЬЯРСКА Иванка (US)
申请号：
RU2013148024/10
公开号：
RU2013148024A
申请日:
2012.03.28
申请国别(地区):
RU
年份:
2015
代理人:
摘要：
1. The double-stranded ribonucleic acid (dsRNA) for inhibiting expression TMPRSS6, wherein said dsRNA comprises a sense strand and an antisense strand, wherein the antisense strand comprises a region of complementarity to the transcript TMPRSS6, comprising at least 15 contiguous nucleotides that differs by no more than 3 nucleotides from the one of the antisense sequences of tables 2, 3, or 4.2. dsRNA according to Claim. 1, wherein said dsRNA comprises at least one modified nukleotid.3. dsRNA according to claim 2, wherein at least one of said modified nucleotide is selected from the group consisting of:. 2-O-methyl modified nucleotide, a nucleotide comprising a 5-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic kisloty.4 bisdetsilamidnoy group. dsRNA according to claim 2, wherein said modified nucleotide is selected from the group consisting of: a. 2-deoxy-2-fluoro-modified nucleotide, 2-deoxy-modified nucleotide, the blocked nucleotide abazicheskogo nucleotide, 2-amino-modified nucleotide, 2-alkyl-modified nucleotide, morfolinonukleotida, phosphoramidate, and a non-natural base comprising nukleotid.5. dsRNA according to Claim. 1, wherein the region of complementarity is composed of at least 17 nucleotides in dlinu.6. dsRNA according to Claim. 1, wherein the complementary region comprises from 19 to 21 nucleotides in dlinu.7. dsRNA according to Claim. 1, wherein the region of complementarity of 19 nucleotides comprising in dlinu.8. dsRNA according to Claim. 1, wherein each chain comprises no more than 30 nucleotides in dlinu.9. dsRNA according to Claim. 1, wherein at least one chain comprises a 3 sticky end, at least one of nukleotida.10. dsRNA according to Claim. 1, r1. Двухцепочечная рибонуклеиновая кислота (dsRNA) для ингибирования экспрессии TMPRSS6, где указанная dsRNA включает смысловую цепь и антисмысловую цепь, причем антисмысловая цепь включает область комплементарности транскрипту TMPRSS6, включающую, по ме
