KHOVOROVA Anastasia,ХОВОРОВА Анастасия,SALOMON William,САЛОМОН Уильям,KAMENS Joanne,КАМЕНС Джоан,SAMARSKY Dmitry,САМАРСКИЙ Дмитрий,WOOLF Tod M.,ВУЛЬФ Тод М.,CARDIA James,КАРДИА Джеймс
申请号：
RU2012144738
公开号：
RU0002615143C2
申请日:
2011.03.24
申请国别(地区):
RU
年份:
2017
代理人:
摘要：
FIELD: biotechnology.SUBSTANCE: invention relates to self-delivered interfering RNA (sd-RNA), which can be used in medicine. Self-delivered interfering sd-RNA contains leading strand and passenger strand, wherein sd-RNA contains double-stranded region and single-stranded region, where single-stranded region is located at 3-end of leading strand, double-stranded region has length of 8–15 nucleotides, and single-stranded region has length of 4–12 nucleotides, at least 40 % of sd-RNA nucleotides are modified, wherein modifications are selected from group containing 5-phosphorylation, phosphorothioate modification of frame, 2-deoxy-, 2F- and 2O-methyl modification of ribose, hydrophobic modification of U and/or C, wherein hydrophobic modification is in position 4 or 5 U and/or C, at least two U and/or C include hydrophobic modification, selected from group consisting of metyl, octyl, thiophene, octyn-1-yl, ethynyl, pyridyl amide, isobutyl, phenyl, 4-pyridyl, 2-pyridyl, indolyl, butyl, naphthyl and imidazole modification single-stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11 or 12 phosphorothioate modifications and modifications include at least one 2F- or 2O-methyl modification sd-RNA optionally contains at least one additional single stranded region and sd-RNA optionally contains cholesterol or long-chain alkylcholesterol analogue attached to 3-end directly or by linker fragment.EFFECT: novel self-delivered interfering RNA and compositions based thereon effective for gene silencing.10 cl, 85 dwg, 1 tbl, 7 exИзобретение относится к самодоставляющейся интерферирующей РНК (sd-РНК), которая может использоваться в медицине. Самодоставляющаяся интерферирующая sd-РНК содержит ведущую нить и пассажирскую нить, причем sd-РНК содержит двухцепочечную область и одноцепочечную область, где одноцепочечная область расположена на 3-конце ведущей нити, двухцепочечная область имеет длину 8-15 нуклеотидов, а одноцепочечная область имеет длину 4-12 нуклеотидов, по меньшей мере 40% ну
