RU2010119292A - Способный к интерференции дуплекс рнк, имеющий "тупые" концы и 3'-модификации - Google Patents

Способный к интерференции дуплекс рнк, имеющий "тупые" концы и 3'-модификации Download PDF

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RU2010119292A
RU2010119292A RU2010119292/10A RU2010119292A RU2010119292A RU 2010119292 A RU2010119292 A RU 2010119292A RU 2010119292/10 A RU2010119292/10 A RU 2010119292/10A RU 2010119292 A RU2010119292 A RU 2010119292A RU 2010119292 A RU2010119292 A RU 2010119292A
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double
stranded rna
rna according
chemical modification
ribonucleotides
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Ян ВАЙЛЕР (DE)
Ян Вайлер
Джонатан ХОЛЛ (CH)
Джонатан Холл
Жан-Шарль БОЛОНЬЯ (CH)
Жан-Шарль БОЛОНЬЯ
Франсуа Жан-Шарль НАТТ (CH)
Франсуа Жан-Шарль НАТТ
Роберт ХЭНЕР (CH)
Роберт ХЭНЕР
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Новартис АГ (CH)
Новартис Аг
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Abstract

1. Двухцепочечная РНК по меньшей мере с одним «тупым» концом, где двухцепочечная РНК включает на 3'-конце по меньшей мере одной цепи химическую модификацию, где химической модификацией является сложный гироксипропилфосфодиэфир. ! 2. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей. ! 3. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает два тупых конца. ! 4. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает два тупых конца и химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей. ! 5. Двухцепочечная РНК по п.1, которая дополнительно включает по меньшей мере один модифицированный рибонуклеотид, содержащий 2'-алкоксильный заместитель. ! 6. Двухцепочечная РНК по п.5, где 2'-алкоксильный заместитель является 2'-O-(2 метоксиэтилом). ! 7. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов. ! 8. Двухцепочечная РНК по п.1, где двухцепочечная РНК имеет длину из 19 смежных пар оснований рибонуклеотидов. ! 9. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов и двухцепочечная РНК включает два тупых конца. ! 10. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов, двухцепочечная РНК включает два тупых конца и химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей. ! 11. Двухцепочечная РНК по п.1, где двухцепочечная РНК имеет длину из 19 смежных пар оснований рибонуклеотидов, двухцепочечная РНК включает два тупых

Claims (15)

1. Двухцепочечная РНК по меньшей мере с одним «тупым» концом, где двухцепочечная РНК включает на 3'-конце по меньшей мере одной цепи химическую модификацию, где химической модификацией является сложный гироксипропилфосфодиэфир.
2. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей.
3. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает два тупых конца.
4. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает два тупых конца и химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей.
5. Двухцепочечная РНК по п.1, которая дополнительно включает по меньшей мере один модифицированный рибонуклеотид, содержащий 2'-алкоксильный заместитель.
6. Двухцепочечная РНК по п.5, где 2'-алкоксильный заместитель является 2'-O-(2 метоксиэтилом).
7. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов.
8. Двухцепочечная РНК по п.1, где двухцепочечная РНК имеет длину из 19 смежных пар оснований рибонуклеотидов.
9. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов и двухцепочечная РНК включает два тупых конца.
10. Двухцепочечная РНК по п.1, где двухцепочечная РНК включает участок из 19 смежных пар оснований рибонуклеотидов, двухцепочечная РНК включает два тупых конца и химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей.
11. Двухцепочечная РНК по п.1, где двухцепочечная РНК имеет длину из 19 смежных пар оснований рибонуклеотидов, двухцепочечная РНК включает два тупых конца и химическую модификацию сложным гироксипропилфосфодиэфиром на 3'-конце обоих цепей.
12. Фармацевтическая композиция, содержащая двухцепочечную РНК по п.1.
13. Двухцепочечная РНК по п.1 для применения в качестве лекарственного средства для селективного ингибирования гена-мишени.
14. Двухцепочечная РНК по п.1 для применения для приготовления лекарственного средства для селективного ингибирования гена-мишени.
15. Двухцепочечная РНК по п.1 для применения для селективного ингибирования гена-мишени in vitro или у животных, кроме человека.
RU2010119292/10A 2003-08-28 2010-05-14 Способный к интерференции дуплекс рнк, имеющий "тупые" концы и 3'-модификации RU2010119292A (ru)

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