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MRNA COMPOSITIONS FOR SIMILAR TO ANGIOPOETIN 3 (ANGPTL3) PROTEIN AND WAYS OF THEIR APPLICATION
专利权人:
ЭЛНИЛЭМ ФАРМАСЬЮТИКАЛЗ (US)
发明人:
БЕТТЕНКОРТ Брайан (US),КУЭРБС Уилльям (US),ФИТЦЖЕРАЛЬД Кевин (US),ФРАНК-КАМЕНЕТСКИ Мария (US),МИЛСТЕЙН Стюарт (US),ШУЛЬГА-МОРСКАЯ Светлана (US)
申请号:
RU2014101627/10
公开号:
RU2014101627A
申请日:
2012.06.20
申请国别(地区):
RU
年份:
2015
代理人:
摘要:
1. Double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of ANGPTL3, wherein said dsRNA contains a sense strand and an antisense strand, wherein said sense strand contains at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO: 1 and said antisense strand contains at least 15 adjacent nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO: 5.2. A double stranded ribonucleic acid (dsRNA) for inhibiting the expression of ANGPTL3, wherein said dsRNA contains a sense strand and an antisense strand, where the antisense strand contains a complementarity site that contains at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from any of the antisense sequences, listed in tables 2, 3, 7, 8, 9, and 10.3. dsRNA according to claim 2, wherein the sense and antisense chains contain sequences selected from the group consisting of AD-52981.1, AD-53063.1, AD-53001.1, AD-53015.1, AD-52986.1, AD-52953.1, AD-53024.1, AD- 53033.1, AD-53030.1, AD-53080.1, AD-53073.1, AD-53132.1, AD-52983.1, AD-52954.1, AD-52961.1, AD-52994.1, AD-52970.1, AD-53075.1, AD-53147.1, AD-53077.1 of tables 7 and 8.4. dsRNA according to claim 1 or 2, wherein said dsRNA contains at least one modified nucleotide. dsRNA according to claim 4, wherein at least one of said modified nucleotides is selected from the group consisting of a 2′-O-methylmodified nucleotide, a nucleotide containing a 5′-thiophosphate group, and a terminal nucleotide linked to a cholesteryl derivative or a dodecanova bisdecylamide group acids. 6. dsRNA according to claim 4, wherein said modified nucleotide is selected from the group consisting of 2′-deoxy-1. Двухцепочечная рибонуклеиновая кислота (дцРНК) для ингибирования экспрессии ANGPTL3, где указанная дцРНК содержит смысловую цепь и антисмысловую цепь, где указанная смысловая цепь содержит по меньшей мере 15 смежных нуклеотидов, отличающихся не б
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