RU2017116544A - Модулирующие полинуклеотиды - Google Patents

Модулирующие полинуклеотиды Download PDF

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RU2017116544A
RU2017116544A RU2017116544A RU2017116544A RU2017116544A RU 2017116544 A RU2017116544 A RU 2017116544A RU 2017116544 A RU2017116544 A RU 2017116544A RU 2017116544 A RU2017116544 A RU 2017116544A RU 2017116544 A RU2017116544 A RU 2017116544A
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loop
stem
modulating polynucleotide
chain
modulating
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RU2017116544A
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Цзиньчжао ХОУ
Матье Э. НОННЕНМАХЕР
Пэнчэн ЧЖОУ
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Вояджер Терапьютикс, Инк.
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Claims (41)

1. Модулирующий полинуклеотид, содержащий
(a) стебель и петлю для образования структуры «петля на стебле», при этом последовательность указанной структуры «петля на стебле» в направлении от 5' до 3' содержит:
(i) мотив UG у основания или возле основания 5'-стебля структуры «петля на стебле»;
(ii) 5'-ветвь стебля, содержащую сопровождающую цепь и необязательно 5'-спейсерный участок, при этом указанный 5'-спейсерный участок, при наличии, расположен между указанным мотивом UG и указанной сопровождающей цепью;
(iii) петлевой участок, содержащий мотив UGUG на 5'-конце указанного петлевого участка;
(iii) 3'-ветвь стебля, содержащую направляющую цепь и необязательно 3'-спейсерный участок, при этом уридин присутствует на 5'-конце направляющей цепи, и при этом указанный 3'-спейсерный участок, при наличии, имеет длину, достаточную для образования одного оборота спирали;
(b) первый фланкирующий участок, расположенный в направлении 5' по отношению к указанной структуре «петля на стебле»; и
(c) второй фланкирующий участок, расположенный в направлении 3' по отношению к указанной структуре «петля на стебле», при этом указанный второй участок содержит мотив CNNC.
2. Модулирующий полинуклеотид по п. 1, где петлевой участок указанной структуры «петля на стебле» получен из канонической микроРНК.
3. Модулирующий полинуклеотид по п. 2, где канонической микроРНК является miR-22.
4. Модулирующий полинуклеотид по п. 3, где по меньшей мере один из указанных первого или второго фланкирующих участков получен из let-7b.
5. Модулирующий полинуклеотид по п. 1, где первым фланкирующим участком является 5'-фланкирующий участок, описанный в таблице 1.
6. Модулирующий полинуклеотид по п. 1, где первым петлевым участком является один из петлевых участков, описанных в таблице 2.
7. Модулирующий полинуклеотид по п. 1, где вторым фланкирующим участком является 3'-фланкирующий участок, описанный в таблице 3.
8. Модулирующий полинуклеотид по п. 1, где направляющая цепь содержит последовательность затравочного участка микроРНК между 2 и 15 положениями.
9. Модулирующий полинуклеотид по п. 8, где направляющая цепь содержит последовательность затравочного участка микроРНК во 2-7, 2-8 или 2-9 положениях.
10. Модулирующий полинуклеотид по п. 1, где длина направляющей цепи составляет 15-30 нуклеотидов.
11. Модулирующий полинуклеотид по п. 10, где сопровождающая цепь по меньшей мере на 70% комплементарна направляющей цепи.
12. Модулирующий полинуклеотид по п. 10, где длина направляющей цепи составляет 21-25 нуклеотидов.
13. Модулирующий полинуклеотид по п. 12, где длина направляющей цепи составляет 22 нуклеотида.
14. Модулирующий полинуклеотид по п. 12, где длина направляющей цепи составляет 21 нуклеотид.
15. Модулирующий полинуклеотид по п. 10, где направляющая цепь по меньшей мере на 70% комплементарна целевой РНК, при этом указанная целевая РНК выбрана из группы, состоящий из кодирующей мРНК млекопитающих и некодирующей РНК млекопитающего.
16. Модулирующий полинуклеотид по п. 15, где целевой РНК является кодирующая мРНК млекопитающего.
17. Модулирующий полинуклеотид по п. 16, где кодирующая мРНК экспрессируется в клетке, ткани или органе нервной системы.
18. Искусственная pre-микроРНК, содержащая
(a) стебель и петлю для образования структуры «петля на стебле», при этом последовательность указанной структуры «петля на стебле» в направлении от 5' до 3' содержит:
(i) 5'-ветвь стебля, содержащую сопровождающую цепь и 5'-спейсерный участок, при этом указанный 5'-спейсерный участок расположен в направлении 5' по отношению к указанной сопровождающей цепи;
(ii) петлевой участок, содержащий мотив UGUG на 5'-конце указанного петлевого участка;
(iii) 3'-ветвь стебля, содержащую направляющую цепь и 3'-спейсерный участок, при этом уридин присутствует на 5'-конце направляющей цепи, и при этом указанный 3'-спейсерный участок имеет длину, достаточную для образования одного оборота спирали; и
(iv) необязательно мотив UG у основания или возле основания 5'-стебля структуры «петля на стебле».
19. Искусственная pre-микроРНК, содержащая
(a) стебель и петлю для образования структуры «петля на стебле», при этом последовательность указанной структуры «петля на стебле» в направлении от 5' до 3' содержит:
(i) 5'-ветвь стебля, содержащую направляющую цепь и 5'-спейсерный участок, при этом указанный 5'-спейсерный участок расположен в направлении 5' по отношению к указанной направляющей цепи;
(ii) петлевой участок, содержащий мотив UGUG на 5'-конце указанного петлевого участка;
(iii) 3'-ветвь стебля, содержащую сопровождающую цепь и 3'-спейсерный участок, при этом уридин присутствует на 5'-конце сопровождающей цепи, и при этом указанный 3'-спейсерный участок имеет длину, достаточную для образования одного оборота спирали; и
(iv) необязательно мотив UG у основания или возле основания 5'-стебля структуры «петля на стебле».
20. Плазмида или вектор, кодирующие модулирующий полинуклеотид по любому из пп. 1-19.
21. Вектор по п. 20, включающий конструкцию на основе рекомбинантного аденоассоциированного вируса (AAV).
22. Рекомбинантный AAV-вирус, включающий конструкцию AAV по п. 21.
23. Рекомбинантный AAV-вирус по п. 22, содержащий капсид серотипа, выбранного из группы, состоящей из AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hu14), AAV10, AAV11, AAV12, AAVrh8, AAVrh10 и AAV-DJ.
24. Рекомбинантный AAV-вирус по п. 23, где капсид серотипа выбран из группы, состоящей из AAV2, AAVrh10, AAV9(hu14), AAV-DJ и AAV9.47.
RU2017116544A 2014-11-14 2015-11-13 Модулирующие полинуклеотиды RU2719192C2 (ru)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462079590P 2014-11-14 2014-11-14
US62/079,590 2014-11-14
US201562212004P 2015-08-31 2015-08-31
US62/212,004 2015-08-31
US201562234477P 2015-09-29 2015-09-29
US62/234,477 2015-09-29
PCT/US2015/060564 WO2016077689A1 (en) 2014-11-14 2015-11-13 Modulatory polynucleotides

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