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GCGR ANTI-SENSE REGULATION
专利权人:
ИНК. (US);АЙСИС ФАРМАСЬЮТИКАЛС
发明人:
ФРЕЙЕР Сьюзан М. (US),БХАНОТ Санжай (US)
申请号:
RU2014115215/10
公开号:
RU2014115215A
申请日:
2012.09.20
申请国别(地区):
RU
年份:
2015
代理人:
摘要:
1. A compound comprising a modified oligonucleotide consisting of 12-30 connected nucleosides and having a nucleobase sequence including a fragment consisting of at least 8 adjacent nucleic bases complementary to an equal length fragment consisting of nucleotides 7267-7283, 7270-7286, 7292-7308, 7295-7311, 7316-7332, 7319-7335, 7341-7357, 7344-7360, 7437-7453, 7365-7381, 7368-7384, 7389-7405, 7392-7408, 7416-7432, 7440- 7456, 7783-7799, 8133-8152, 8144-8160, 9804-9823, 10718-10734 or 15743-15762 of SEQ ID NO: 2, wherein the target of said modified oligonucleotide is SEQ ID NO: 2.2. A compound according to claim 1, characterized in that it consists of a single-chain modified oligonucleotide. A compound according to claim 1, characterized in that said modified oligonucleotide is at least 96% complementary to SEQ ID NO: 2.4. A compound according to claim 1, characterized in that said modified oligonucleotide is at least 97% complementary to SEQ ID NO: 2.5. A compound according to claim 1, characterized in that said modified oligonucleotide is at least 98% complementary to SEQ ID NO: 2.6. A compound according to claim 1, characterized in that said modified oligonucleotide is at least 99% complementary to SEQ ID NO: 2.7. The compound of claim 1, wherein said modified oligonucleotide is 100% complementary to SEQ ID NO: 2.8. A compound according to claim 1, characterized in that at least one internucleoside bond is a modified internucleoside bond. A compound according to claim 8, characterized in that each internucleoside bond is a phosphorothioate internucleoside bond. A compound according to claim 1, characterized in that at least one nucleoside of said modified oligonucleot1. Соединение, включающее модифицированный олигонуклеотид, состоящий из 12-30 соединенных нуклеозидов и обладающий последовательностью нуклеиновых оснований, включающей фрагмент, состоящий из по меньшей мере 8 смежных нуклеиновых оснований, комплементарный фрагменту равной длины, состоящему из нуклеот
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