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RU2018119315A - Олигонуклеотиды для индуцирования отцовской экспрессии ube3a - Google Patents

Олигонуклеотиды для индуцирования отцовской экспрессии ube3a Download PDF

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Publication number
RU2018119315A
RU2018119315A RU2018119315A RU2018119315A RU2018119315A RU 2018119315 A RU2018119315 A RU 2018119315A RU 2018119315 A RU2018119315 A RU 2018119315A RU 2018119315 A RU2018119315 A RU 2018119315A RU 2018119315 A RU2018119315 A RU 2018119315A
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Russia
Prior art keywords
oligonucleotide
oligonucleotide according
paragraphs
conjugate
expression
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RU2018119315A
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English (en)
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RU2018119315A3 (ru
RU2742007C2 (ru
Inventor
Вероника КОСТА
Май Хедтьорн
Мариус Хёнер
Рави Джагасиа
Мадс Аабоэ ЕНСЕН
Кристоф ПАЧ
Люкке ПЕДЕРСЕН
Сёрен Вестергорд РАСМУССЕН
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Ф. Хоффманн-Ля Рош Аг
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Claims (23)

1. Антисмысловой олигонуклеотид, который содержит непрерывную нуклеотидную последовательность из 10-30 нуклеотидов в длину с по меньшей мере 98%-ной комплементарностью относительно положения 30654-30745 в SEQ ID NO: 1 (область A 693 в Таблице 1) или положения 11482-11535 в SEQ ID NO: 1 (Область A321 в Таблице 1).
2. Олигонуклеотид по п. 1, где данный олигонуклеотид содержит последовательность, выбранную из группы, состоящей из SEQ ID NO: 46, 47, 48, 221, 222, 223, 224, 225, 289, 290, 291, 292, 293, 294, 295, 296, 297, 298, 299, 300, 301, 302, 303, 304, 304, 305, 585, 586, 587, 588, 589, 625, 626, 627, 628, 629, 630, 631, 632, 698, 699, 700, 702, 703, 704, 705, 706, 707, 708, 709, 710, 711, 712, 713, 714, 715, 716, 717 и 718, 733, 734, 735, 736, 737, 738, 739, 740, 741, 742, 743, 744, 745, 746, 747, 748, 749, 750, 751, 752, 754, 755, 756, 757, 758, 759, 760, 761, 762, 763, 764, 765, 766, 767, 768, 769, 770, 772, 773, 774, 775, 776, 777, 778, 779, 780, 781, 782, 783, 784, 785, 786, 787, 788, 789, 790, 791, 792, 793, 794, 795, 796, 797, 798, 799, 800, 801, 802, 803, 804, 805, 806, 807.
3. Олигонуклеотид по п. 1 или 2, содержащий или состоящий из последовательности SEQ ID NO: 626.
4. Олигонуклеотид по п. 1 или 2, содержащий или состоящий из последовательности SEQ ID NO: 585.
5. Олигонуклеотид по любому из пп. 1-3, содержащий один или более чем один нуклеозид, модифицированный 2'-сахаром.
6. Олигонуклеотид по п. 5, в котором один или более чем один нуклеозид, модифицированный 2'-сахаром, независимо выбран из группы, состоящей из 2'-O-алкил-РНК, 2'-O-метил-РНК, 2'-O-алкокси-РНК, 2'-O-метоксиэтил-РНК, 2'-амино-ДНК, 2'-фтор-ДНК, арабинонуклеиновой кислоты (ANA), 2'-фтор-ANA и нуклеозидов LNA (запертая нуклеиновая кислота).
7. Олигонуклеотид по п. 5 или 6, в котором один или более чем один модифицированный нуклеозид представляет собой нуклеозид LNA.
8. Олигонуклеотид по любому из пп. 1-7, где данный олигонуклеотид содержит по меньшей мере одну модифицированную межнуклеозидную связь.
9. Олигонуклеотид по п. 8, в котором межнуклеозидные связи в пределах непрерывной нуклеотидной последовательности представляют собой фосфоротиоатные межнуклеозидные связи.
10. Олигонуклеотид по пп. 1-9, где данный олигонуклеотид способен рекрутировать РНКазу Н.
11. Олигонуклеотид по п. 10, где данный олигонуклеотид представляет собой гэпмер (gapmer).
12. Олигонуклеотид по любому из пп. 1-11, где данный олигонуклеотид выбран из группы, состоящей из CMP ID NO: 46_1, 47_1, 48_1, 48_2, 48_3, 48_4, 48_5, 48_6, 48_7, 221_1, 222_1, 223_1, 224_1, 225_1, 289_1, 290_1, 291_1, 292_1, 293_1, 294_1, 295_1, 296_1, 297_1, 298_1, 299_1, 300_1, 301_1, 302_1, 303_1, 304_1, 304_10, 304_2, 304_3, 304_4, 304_5, 304_6, 304_7, 304_8, 304_9, 304_11, 304_12, 304_13, 304_14, 304_15 и 305_1, 585_1, 585_2, 585_3, 585_4, 585_5, 585_6, 585_7, 585_8, 585_9, 585_10, 585_11, 585_12, 585_13, 585_14, 586_1, 586_2, 586_3, 586_4, 586_5, 586_6, 586_7, 586_8, 586_9, 586_10, 586_11, 586_12, 586_13, 586_14, 587_1, 587_2, 587_3, 587_4, 587_5, 587_6, 587_7, 587_8, 587_9, 587_10, 587_11, 587_12, 587_13, 587_14, 588_1, 588_2, 588_3, 588_4, 588_5, 588_6, 588_7, 588_8, 588_9, 588_10, 588_11, 588_12, 588_13, 588_14, 589_1, 589_2, 589_3, 589_4, 589_5, 589_6, 589_7, 589_8, 589_9, 589_10, 589_11, 589_12, 589_13, 589_14, 625_1, 625_2, 625_3, 625_4, 625_5, 625_6, 625_7, 625_8, 625_9, 625_10, 625_11, 625_12, 625_13, 625_14, 626_1, 626_2, 626_3, 626_4, 626_5, 626_6, 626_7, 626_8, 626_9, 626_10, 626_11, 626_12, 626_13, 626_14, 627_1, 627_2, 627_3, 627_4, 627_5, 627_6, 627_7, 627_8, 627_9, 627_10, 627_11, 627_12, 627_13, 627_14, 628_1, 628_2, 628_3, 628_4, 628_5, 628_6, 628_7, 628_8, 628_9, 628_10, 628_11, 628_12, 628_13, 628_14, 629_1, 629_10, 629_11, 629_2, 629_3, 629_4, 629_5, 629_6, 629_7, 629_8, 629_9, 629_12, 629_13, 629_14, 629_15, 629_16, 630_1, 630_2, 630_3, 631_1, 631_10, 631_2, 631_3, 631_4, 631_5, 631_6, 631_7, 631_8, 631_9, 631_11, 631_12, 631_13, 631_14, 631_15, 632_1, 632_2, 632_3, 632_4, 632_5, 632_6, 632_7, 632_8, 632_9, 632_10, 632_11, 632_12, 632_13, 632_14, 698_1, 698_2, 698_3, 698_4, 698_5, 699_1, 699_2, 699_3, 699_4, 699_5, 700_1, 700_2, 700_3, 700_4, 700_5, 701_1, 701_2, 701_3, 701_4, 701_5, 702_1, 702_2, 702_3, 702_4, 702_5, 703_1, 703_2, 703_3, 703_4, 703_5, 704_1, 704_2, 704_3, 704_4, 704_5, 705_1, 705_2, 705_3, 705_4, 705_5, 706_1, 706_2, 706_3, 706_4, 706_5, 707_1, 707_2, 707_3, 707_4, 707_5, 708_1, 708_2, 708_3, 708_4, 708_5, 709_1, 709_2, 709_3, 709_4, 709_5, 710_1, 710_2, 710_3, 710_4, 710_5, 711_1, 711_2, 711_3, 711_4, 711_5, 712_1, 712_2, 712_3, 712_4, 712_5, 713_1, 713_2, 713_3, 713_4, 713_5, 714_1, 714_2, 714_3, 714_4, 714_5, 715_1, 715_2, 715_3, 715_4, 715_5, 716_1, 716_2, 716_3, 716_4, 716_5, 717_1, 717_2, 717_3, 717_4, 717_5, 718_1, 718_2.
13. Олигонуклеотид по п. 12, содержащий или состоящий из CMP ID NO: 626_7.
14. Олигонуклеотид по п. 12, содержащий или состоящий из CMP ID NO: 585_1.
15. Конъюгат, содержащий олигонуклеотид по любому из пп. 1-14 и по меньшей мере одну конъюгатную группировку, ковалентно присоединенную к указанному олигонуклеотиду.
16. Фармацевтическая композиция, содержащая олигонуклеотид по любому из пп. 1-14 или конъюгат по п. 15 и фармацевтически приемлемый разбавитель, растворитель, носитель, соль и/или адъювант.
17. Способ in vivo или in vitro индуцирования экспрессии UBE3A в клетке-мишени, где подавляется экспрессия отцовского UBE3A, причем указанный способ включает введение в указанную клетку олигонуклеотида по любому из пп. 1-14 или конъюгата по п. 15, или фармацевтической композиции по п. 16 в эффективном количестве.
18. Способ по п. 17, в котором экспрессия UBE3A увеличивается по меньшей мере на 40% по сравнению с контролем.
19. Способ по п. 17 или 18, в котором уровень транскрипта SNHG14 ниже SNORD109B уменьшается по меньшей мере на 30% по сравнению с контролем.
20. Способ по пп. 17-19, в котором клетка-мишень представляет собой нейрон.
21. Способ по любому из пп. 17-20, в котором экспрессия SNORD115 не подвергается значимому влиянию по сравнению с контролем.
22. Применение олигонуклеотида по любому из пп. 1-14 или конъюгат по п. 15, или фармацевтическая композиция по п. 16 в лечении или предупреждении синдрома Ангельмана.
23. Применение олигонуклеотида по пп. 1-14 или конъюгата по п. 15, или фармацевтической композиции по п. 16 для приготовления лекарственного средства для лечения или предупреждения синдрома Ангельмана.
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