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RU2019100094A - МОЛЕКУЛЫ НУКЛЕИНОВЫХ КИСЛОТ ДЛЯ УМЕНЬШЕНИЯ УРОВНЯ мРНК PAPD5 ИЛИ PAPD7 ДЛЯ ЛЕЧЕНИЯ ИНФЕКЦИОННОГО ГЕПАТИТА В - Google Patents

МОЛЕКУЛЫ НУКЛЕИНОВЫХ КИСЛОТ ДЛЯ УМЕНЬШЕНИЯ УРОВНЯ мРНК PAPD5 ИЛИ PAPD7 ДЛЯ ЛЕЧЕНИЯ ИНФЕКЦИОННОГО ГЕПАТИТА В Download PDF

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RU2019100094A
RU2019100094A RU2019100094A RU2019100094A RU2019100094A RU 2019100094 A RU2019100094 A RU 2019100094A RU 2019100094 A RU2019100094 A RU 2019100094A RU 2019100094 A RU2019100094 A RU 2019100094A RU 2019100094 A RU2019100094 A RU 2019100094A
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nucleic acid
acid molecule
papd5
inhibits
composition
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RU2019100094A
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RU2768699C2 (ru
RU2019100094A3 (ru
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Хассан ЯВАНБАКХТ
Хенрик МЮЛЛЕР
Сёрен Оттосен
Люкке ПЕДЕРСЕН
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Ф. Хоффманн-Ля Рош Аг
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Claims (37)

1. Композиция, содержащая молекулу нуклеиновой кислоты, для применения в лечении и/или предупреждении инфекции вирусом гепатита В, где указанная молекула нуклеиновой кислоты ингибирует экспрессию и/или активность PAPD5.
2. Композиция для применения по п. 1, где указанная композиция представляет собой комбинированный препарат, содержащий:
а. молекулу нуклеиновой кислоты, которая ингибирует экспрессию и/или активность PAPD5; и
б. молекулу нуклеиновой кислоты, которая ингибирует экспрессию и/или активность PAPD7.
3. Композиция для применения по п. 1 или 2, где молекулы нуклеиновой кислоты независимо выбраны из группы, состоящей из следующих:
а. одноцепочечный антисмысловой олигонуклеотид;
б. молекула миРНК (малая интерферирующая РНК);
в. молекула кшРНК (короткая шпилечная РНК); и
г. аппарат редактирования генома, содержащий:
i. сайт-специфичную ДНК-нуклеазу или полинуклеотид, кодирующий сайт-специфичную ДНК-нуклеазу; и
ii. РНК-проводник или полинуклеотид, кодирующий РНК-проводник.
4. Композиция для применения по п. 2 или 3, где молекулы нуклеиновой кислоты выбраны из следующих:
а. одноцепочечный антисмысловой олигонуклеотид, содержащий непрерывную нуклеотидную последовательность из 10-30 нуклеотидов в длину с по меньшей мере 80%-ной комплементарностью с нуклеиновой кислотой-мишенью PAPD5, и который способен уменьшать экспрессию PAPD5; и
б. одноцепочечный антисмысловой олигонуклеотид, содержащий непрерывную нуклеотидную последовательность из 10-30 нуклеотидов в длину с по меньшей мере 80%-ной комплементарностью с нуклеиновой кислотой-мишенью PAPD7, и который способен уменьшать экспрессию PAPD7.
5. Композиция для применения по любому из пп. 1-4, где данная композиция уменьшает секрецию HBsAg, HBeAg и/или ингибирует продукцию внутриклеточной мPHK HBV или ДНК HBV.
6. Композиция для применения по любому из пп. 1-5, где данная композиция ингибирует развитие хронической инфекции HBV и/или уменьшает зараженность человека, инфицированного HBV.
7. Способ идентификации соединения или композиции, которая предупреждает, уменьшает интенсивность и/или ингибирует инфекцию вирусом гепатита В (HBV), включающий:
а. приведение в контакт тестируемого соединения или композиции с клеткой, экспрессирующей PAPD5 и/или PAPD7;
б. измерение экспрессии и/или активности PAPD5 и/или PAPD7 в присутствии и в отсутствие указанного тестируемого соединения или композиции; и
в. идентификацию соединения или композиции, которая уменьшает экспрессию и/или активность PAPD5 и/или PAPD7, в качестве соединения, которое предупреждает, уменьшает интенсивность и/или ингибирует инфекцию HBV.
8. Способ по п. 7, в котором тестируемое соединение представляет собой библиотеку молекул нуклеиновой кислоты, и на стадии в) идентифицируются молекулы нуклеиновой кислоты, которые уменьшают экспрессию мРНК PAPD5 или PAPD7 по меньшей мере на 60%.
9. Способ по п. 7, в котором тестируемое соединение представляет собой комбинированный препарат молекулы нуклеиновой кислоты, способной уменьшать уровень PAPD5, и молекулы нуклеиновой кислоты, способной уменьшать уровень PAPD7.
10. Способ по любому из пп. 7-9, в котором соединение, которое ингибирует размножение HBV, ингибирует секрецию антигена поверхности HBV (HBsAg) и/или ингибирует секрецию антигена оболочки HBV (НВеАg), и/или ингибирует образование внутриклеточной мРНК HBV.
11. Одноцепочечный антисмысловой олигонуклеотид, который содержит или состоит из непрерывной нуклеотидной последовательности из 10-30 нуклеотидов в длину, где данная непрерывная нуклеотидная последовательность является на 100% комплементарной нуклеиновой кислоте-мишени PAPD5, и данный антисмысловой олигонуклеотид способен уменьшать экспрессию PAPD5.
12. Молекула нуклеиновой кислоты, которая содержит или состоит из непрерывной нуклеотидной последовательности из 10-30 нуклеотидов в длину, где данная непрерывная нуклеотидная последовательность является на 100% комплементарной нуклеиновой кислоте-мишени PAPD7, и данная молекула нуклеиновой кислоты способна уменьшать экспрессию PAPD7.
13. Молекула нуклеиновой кислоты по п. 12, где данная молекула нуклеиновой кислоты представляет собой одноцепочечный антисмысловой олигонуклеотид.
14. Антисмысловой олигонуклеотид или молекула нуклеиновой кислоты по любому из пп. 11-13, содержащая один или более чем один нуклеозид, модифицированный 2' сахаром.
15. Антисмысловой олигонуклеотид или молекула нуклеиновой кислоты по п. 14, где один или более чем один нуклеозид, модифицированный 2' сахаром, независимо выбран из группы, состоящей из 2'-O-алкил-РНК, 2'-O-метил-РНК, 2'-алкокси-РНК, 2'-O-метоксиэтил-РНК, 2'-амино-ДНК, 2'-фтор-ДНК, арабинонуклеиновой кислоты (ANA), 2'-фтор-ANA и нуклеозидов LNA.
16. Антисмысловой олигонуклеотид или молекула нуклеиновой кислоты по п. 14 или 15, где один или более чем один нуклеозид, модифицированный 2' сахаром, представляет собой нуклеозид LNA.
17. Антисмысловой олигонуклеотид или молекула нуклеиновой кислоты по любому из пп. 11-16, где межнуклеозидные связи в пределах непрерывной нуклеотидной последовательности представляют собой фосфоротиоатные межнуклеозидные связи.
18. Антисмысловой олигонуклеотид по любому из пп. 11 или 13-17, где данный олигонуклеотид представляет собой гэпмер формулы 5'-F-G-F'-3', где область F и F' независимо содержит 1-7 нуклеозидов, модифицированных 2' сахаром, и G представляет собой область из 6-16 нуклеозидов, которые способны рекрутировать РНКазуН.
19. Конъюгат, содержащий антисмысловой олигонуклеотид или молекулу нуклеиновой кислоты по любому из пп. 11-18 и по меньшей мере одну конъюгатную группировку, ковалентно присоединенную к указанному олигонуклеотиду.
20. Комбинированный препарат, содержащий:
а. молекулу нуклеиновой кислоты, которая ингибирует экспрессию и/или активность PAPD5; и
б. молекулу нуклеиновой кислоты, которая ингибирует экспрессию и/или активность PAPD7.
21. Комбинированный препарат по п. 20, в котором молекула нуклеиновой кислоты выбрана из любой молекулы нуклеиновой кислоты по пп. 11-18 или конъюгата по п. 19.
22. Фармацевтическая композиция, содержащая антисмысловой олигонуклеотид или молекулу нуклеиновой кислоты по любому из пп. 11-18, или конъюгат по п. 19, или комбинированный препарат по п. 20 или 21.
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