| abstract |
FIELD: biotechnology. n 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-, 2'F- and 2'O-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 2'F- or 2'O-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. n EFFECT: novel self-delivered interfering RNA and compositions based thereon effective for gene silencing. n 10 cl, 85 dwg, 1 tbl, 7 ex |