RU2018140495A - Модулирующие полинуклеотиды - Google Patents

Модулирующие полинуклеотиды Download PDF

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RU2018140495A
RU2018140495A RU2018140495A RU2018140495A RU2018140495A RU 2018140495 A RU2018140495 A RU 2018140495A RU 2018140495 A RU2018140495 A RU 2018140495A RU 2018140495 A RU2018140495 A RU 2018140495A RU 2018140495 A RU2018140495 A RU 2018140495A
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Динах Вен-Йи Сах
Цзиньчжао ХОУ
Матье НОННЕНМАХЕР
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Вояджер Терапьютикс, Инк.
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Claims (46)

1. Модулирующий полинуклеотид, содержащий
(a) стебель и петлю, образующую структуру «стебель-петля», последовательность указанной структуры «стебель-петля» от 5' к 3' содержит:
(i) стебель на 5'-конце, содержащий сопровождающую цепь;
(ii) область петли, где область петли содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:16, 10, 11, 12, 13, 14, 15, 17, 18 и 19;
(iii) плечо стебля на 3'-конце, содержащее направляющую цепь, где уридин присутствует на 5'-конце направляющей цепи;
(b) первую фланкирующую область, расположенную в направлении 5' от указанной структуры «стебель-петля»,
где первая фланкирующая область содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:5, 1, 2, 3, 4, 6, 7, 8 и 9; и
(c) вторую фланкирующую область, расположенную в направлении 3' от указанной структуры «стебель-петля»,
где указанная вторая фланкирующая область содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:21, 20, 22, 23, 24, 25, 26 и 27.
2. Модулирующий полинуклеотид, содержащий
(a) стебель и петлю, образующие структуру «стебель-петля», последовательность указанной структуры «стебель-петля» от 5' к 3' содержит:
(i) плечо стебля на 5’-конце, содержащее направляющую цепь, где уридин присутствует на 5'-конце направляющей цепи;
(ii) область петли, где область петли содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:16, 10, 11, 12, 13, 14, 15, 17, 18 и 19;
(iii) плечо стебля на 3’-конце, содержащее сопровождающую цепь;
(b) первую фланкирующую область, расположенную в направлении 5' от указанной структуры «стебель-петля»,
где первая фланкирующая область содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:5, 1, 2, 3, 4, 6, 7, 8 и 9; и
(c) вторую фланкирующую область, расположенную в направлении 3' от указанной структуры «стебель-петля»,
где указанная вторая фланкирующая область содержит последовательность, выбранную из группы, состоящей из SEQ ID NO:21, 20, 22, 23, 24, 25, 26 и 27.
3. Модулирующий полинуклеотид по п.1 или 2, в котором область петли указанной структуры «стебель-петля» получают из канонической микроРНК.
4. Модулирующий полинуклеотид по п.3, в котором каноническая микроРНК представляет собой miR-22.
5. Модулирующий полинуклеотид по п.4, в котором по меньшей мере одну из указанных первой или второй фланкирующих областей получают из let-7b.
6. Модулирующий полинуклеотид по п.1 или 2, в котором область петли содержит SEQ ID NO:16.
7. Модулирующий полинуклеотид по п.1 или 2, в котором первая фланкирующая область содержит SEQ ID NO:5.
8. Модулирующий полинуклеотид по п.1 или 2, в котором вторая фланкирующая область содержит SEQ ID NO:21.
9. Модулирующий полинуклеотид по п.1 или 2, в котором структура «стебель-петля» содержит мотив UG внути или около основания стебля на 5’-конце структуры «стебель-петля».
10. Модулирующий полинуклеотид по п.1 или 2, в котором область петли содержит мотив UGUG на 5'-конце указанной области петли.
11. Модулирующий полинуклеотид по п.1 или 2, в котором вторая область содержит мотив CNNC.
12. Модулирующий полинуклеотид по п.9, в котором плечо на 5'-конце содержит спейсерную область на 5’-конце, расположенную между мотивом UG и сопровождающей цепью или направляющей цепью.
13. Модулирующий полинуклеотид по п.1 или 2, в котором плечо стебля на 3’-конце содержит 3' спейсерную область,
где спейсерная область на 3’-конце имеет длину, достаточную для формирования одного оборота спирали.
14. Модулирующий полинуклеотид по п.1 или 2, в котором направляющая цепь содержит затравочную последовательность микроРНК между положениями 2 и 9.
15. Модулирующий полинуклеотид по п.1 или 2, в котором направляющая цепь содержит затравочную последовательность микроРНК между положениями 2 и 8.
16. Модулирующий полинуклеотид по п.1 или 2, в котором направляющая цепь содержит затравочную последовательность микроРНК между положениями 2 и 7.
17. Модулирующий полинуклеотид по п.1 или 2, в котором направляющая цепь составляет 15-30 нуклеотидов в длину.
18. Модулирующий полинуклеотид по п.17, в котором направляющая цепь составляет 21-25 нуклеотидов в длину.
19. Модулирующий полинуклеотид по п.18, в котором направляющая цепь составляет 22 нуклеотида в длину.
20. Модулирующий полинуклеотид по п.18, в котором направляющая цепь составляет 21 нуклеотид в длину.
21. Модулирующий полинуклеотид по п.17, в котором сопровождающая цепь по меньшей мере на 70% комплементарна направляющей цепи.
22. Модулирующий полинуклеотид по п.17, в котором направляющая цепь по меньшей мере на 60% комплементарна целевой РНК,
где указанная целевая РНК выбрана из группы, состоящей из кодирующей мРНК млекопитающего и не кодирующей РНК млекопитающего.
23. Модулирующий полинуклеотид по п.22, в котором целевая РНК представляет собой кодирующую мРНК млекопитающего.
24. Модулирующий полинуклеотид по п.23, в котором кодирующую мРНК экспрессируют в неврологической клетке, ткани или органе.
25. Плазмида или вектор, кодирующие модулирующий полинуклеотид по любому из пп.1-24.
26. Вектор по п.25, содержащий рекомбинантную аденоассоциированную вирусную (AAV) конструкцию.
27. Рекомбинантный AAV вирус, содержащий AAV конструкцию по п.26.
28. Рекомбинантный AAV вирус по п.27, который имеет серотип капсида, выбранный из группы, состоящей из AAV1, AAV2, AAV2G9, AAV3, AAV3a, AAV3b, AAV3-3, AAV4, AAV4-4, AAV5, AAV6, AAV6.1, AAV6.2, AAV6.1.2, AAV7, AAV7.2, AAV8, AAV9, AAV9.11, AAV9.13, AAV9.16, AAV9.24, AAV9.45, AAV9.47, AAV9.61, AAV9.68, AAV9.84, AAV9.9, AAV10, AAV11, AAV12, AAV16.3, AAV24.1, AAV27.3, AAV42.12, AAV42-1b, AAV42-2, AAV42-3a, AAV42-3b, AAV42-4, AAV42-5a, AAV42-5b, AAV42-6b, AAV42-8, AAV42-10, AAV42-11, AAV42-12, AAV42-13, AAV42-15, AAV42-aa, AAV43-1, AAV43-12, AAV43-20, AAV43-21, AAV43-23, AAV43-25, AAV43-5, AAV44.1, AAV44.2, AAV44.5, AAV223.1, AAV223.2, AAV223.4, AAV223.5, AAV223.6, AAV223.7, AAV1-7/rh.48, AAV1-8/rh.49, AAV2-15/rh.62, AAV2-3/rh.61, AAV2-4/rh.50, AAV2-5/rh.51, AAV3.1/hu.6, AAV3.1/hu.9, AAV3-9/rh.52, AAV3-11/rh.53, AAV4-8/r11.64, AAV4-9/rh.54, AAV4-19/rh.55, AAV5-3/rh.57, AAV5-22/rh.58, AAV7.3/hu.7, AAV16.8/hu.10, AAV16.12/hu.11, AAV29.3/bb.1, AAV29.5/bb.2, AAV106.1/hu.37, AAV114.3/hu.40, AAV127.2/hu.41, AAV127.5/hu.42, AAV128.3/hu.44, AAV130.4/hu.48, AAV145.1/hu.53, AAV145.5/hu.54, AAV145.6/hu.55, AAV161.10/hu.60, AAV161.6/hu.61, AAV33.12/hu.17, AAV33.4/hu.15, AAV33.8/hu.16, AAV52/hu.19, AAV52.1/hu.20, AAV58.2/hu.25, AAVA3.3, AAVA3.4, AAVA3.5, AAVA3.7, AAVC1, AAVC2, AAVC5, AAV-DJ, AAV-DJ8, AAVF3, AAVF5, AAVH2, AAVrh.72, AAVhu.8, AAVrh.68, AAVrh.70, AAVpi.1, AAVpi.3, AAVpi.2, AAVrh.60, AAVrh.44, AAVrh.65, AAVrh.55, AAVrh.47, AAVrh.69, AAVrh.45, AAVrh.59, AAVhu.12, AAVH6, AAVLK03, AAVH-1/hu.1, AAVH-5/hu.3, AAVLG-10/rh.40, AAVLG-4/rh.38, AAVLG-9/hu.39, AAVN721-8/rh.43, AAVCh.5, AAVCh.5R1, AAVcy.2, AAVcy.3, AAVcy.4, AAVcy.5, AAVCy.5R1, AAVCy.5R2, AAVCy.5R3, AAVCy.5R4, AAVcy.6, AAVhu.1, AAVhu.2, AAVhu.3, AAVhu.4, AAVhu.5, AAVhu.6, AAVhu.7, AAVhu.9, AAVhu.10, AAVhu.11, AAVhu.13, AAVhu.15, AAVhu.16, AAVhu.17, AAVhu.18, AAVhu.20, AAVhu.21, AAVhu.22, AAVhu.23.2, AAVhu.24, AAVhu.25, AAVhu.27, AAVhu.28, AAVhu.29, AAVhu.29R, AAVhu.31, AAVhu.32, AAVhu.34, AAVhu.35, AAVhu.37, AAVhu.39, AAVhu.40, AAVhu.41, AAVhu.42, AAVhu.43, AAVhu.44, AAVhu.44R1, AAVhu.44R2, AAVhu.44R3, AAVhu.45, AAVhu.46, AAVhu.47, AAVhu.48, AAVhu.48R1, AAVhu.48R2, AAVhu.48R3, AAVhu.49, AAVhu.51, AAVhu.52, AAVhu.54, AAVhu.55, AAVhu.56, AAVhu.57, AAVhu.58, AAVhu.60, AAVhu.61, AAVhu.63, AAVhu.64, AAVhu.66, AAVhu.67, AAVhu.14/9, AAVhu.t 19, AAVrh.2, AAVrh.2R, AAVrh.8, AAVrh.8R, AAVrh.10, AAVrh.12, AAVrh.13, AAVrh.13R, AAVrh.14, AAVrh.17, AAVrh.18, AAVrh.19, AAVrh.20, AAVrh.21, AAVrh.22, AAVrh.23, AAVrh.24, AAVrh.25, AAVrh.31, AAVrh.32, AAVrh.33, AAVrh.34, AAVrh.35, AAVrh.36, AAVrh.37, AAVrh.37R2, AAVrh.38, AAVrh.39, AAVrh.40, AAVrh.46, AAVrh.48, AAVrh.48.1, AAVrh.48.1.2, AAVrh.48.2, AAVrh.49, AAVrh.51, AAVrh.52, AAVrh.53, AAVrh.54, AAVrh.56, AAVrh.57, AAVrh.58, AAVrh.61, AAVrh.64, AAVrh.64R1, AAVrh.64R2, AAVrh.67, AAVrh.73, AAVrh.74, AAVrh8R, мутант A586R AAVrh8R, R533A мутант AAVrh8R, AAAV, BAAV, AAV козы, AAV коровы, AAVhE1.1, AAVhEr1.5, AAVhER1.14, AAVhEr1.8, AAVhER1.16, AAVhER1.18, AAVhEr1.35, AAVhEr1.7, AAVhEr1.36, AAVhEr2.29, AAVhEr2.4, AAVhEr2.16, AAVhEr2.30, AAVhEr2.31, AAVhEr2.36, AAVhER1.23, AAVhEr3.1, AAV2.5T, AAV-PAEC, AAV-LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV-LK07, AAV-LK08, AAV-LK09, AAV-LK10, AAV-LK11, AAV-LK12, AAV-LK13, AAV-LK14, AAV-LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2, AAV-PAEC4, AAV-PAEC6, AAV-PAEC7, AAV-PAEC8, AAV-PAEC11, AAV-PAEC12, AAV-2-пре-мкРНК-101, AAV-8h, AAV-8b, AAV-h, AAV-b, AAV SM 10-2, AAV Shuffle 100-1, AAV Shuffle 100-3, AAV Shuffle 100-7, AAV Shuffle 10-2, AAV Shuffle 10-6, AAV Shuffle 10-8, AAV Shuffle 100-2, AAV SM 10-1, AAV SM 10-8, AAV SM 100-3, AAV SM 100-10, BNP61 AAV, BNP62 AAV, BNP63 AAV, AAVrh.50, AAVrh.43, AAVrh.62, AAVrh.48, AAVhu.19, AAVhu.11, AAVhu.53, AAV4-8/rh.64, AAVLG-9/hu.39, AAV54.5/hu.23, AAV54.2/hu.22, AAV54.7/hu.24, AAV54.1/hu.21, AAV54.4R/hu.27, AAV46.2/hu.28, AAV46.6/hu.29, AAV128.1/hu.43, AAV подлинного типа (ttAAV), UPENN AAV 10, японский AAV 10, AAV CBr-7.1, AAV CBr-7.10, AAV CBr-7.2, AAV CBr-7.3, AAV CBr-7.4, AAV CBr-7.5, AAV CBr-7.7, AAV CBr-7.8, AAV CBr-B7.3, AAV CBr-B7.4, AAV CBr-E1, AAV CBr-E2, AAV CBr-E3, AAV CBr-E4, AAV CBr-E5, AAV CBr-e5, AAV CBr-E6, AAV CBr-E7, AAV CBr-E8, AAV CHt-1, AAV CHt-2, AAV CHt-3, AAV CHt-6.1, AAV CHt-6.10, AAV CHt-6.5, AAV CHt-6.6, AAV CHt-6.7, AAV CHt-6.8, AAV CHt-P1, AAV CHt-P2, AAV CHt-P5, AAV CHt-P6, AAV CHt-P8, AAV CHt-P9, AAV CKd-1, AAV CKd-10, AAV CKd-2, AAV CKd-3, AAV CKd-4, AAV CKd-6, AAV CKd-7, AAV CKd-8, AAV CKd-B1, AAV CKd-B2, AAV CKd-B3, AAV CKd-B4, AAV CKd-B5, AAV CKd-B6, AAV CKd-B7, AAV CKd-B8, AAV CKd-H1, AAV CKd-H2, AAV CKd-H3, AAV CKd-H4, AAV CKd-H5, AAV CKd-H6, AAV CKd-N3, AAV CKd-N4, AAV CKd-N9, AAV CLg-F1, AAV CLg-F2, AAV CLg-F3, AAV CLg-F4, AAV CLg-F5, AAV CLg-F6, AAV CLg-F7, AAV CLg-F8, AAV CLv-1, AAV CLv1-1, AAV Clv1-10, AAV CLv1-2, AAV CLv-12, AAV CLv1-3, AAV CLv-13, AAV CLv1-4, AAV Clv1-7, AAV Clv1-8, AAV Clv1-9, AAV CLv-2, AAV CLv-3, AAV CLv-4, AAV CLv-6, AAV CLv-8, AAV CLv-D1, AAV CLv-D2, AAV CLv-D3, AAV CLv-D4, AAV CLv-D5, AAV CLv-D6, AAV CLv-D7, AAV CLv-D8, AAV CLv-E1, AAV CLv-K1, AAV CLv-K3, AAV CLv-K6, AAV CLv-L4, AAV CLv-L5, AAV CLv-L6, AAV CLv-M1, AAV CLv-M11, AAV CLv-M2, AAV CLv-M5, AAV CLv-M6, AAV CLv-M7, AAV CLv-M8, AAV CLv-M9, AAV CLv-R1, AAV CLv-R2, AAV CLv-R3, AAV CLv-R4, AAV CLv-R5, AAV CLv-R6, AAV CLv-R7, AAV CLv-R8, AAV CLv-R9, AAV CSp-1, AAV CSp-10, AAV CSp-11, AAV CSp-2, AAV CSp-3, AAV CSp-4, AAV CSp-6, AAV CSp-7, AAV CSp-8, AAV CSp-8.10, AAV CSp-8.2, AAV CSp-8.4, AAV CSp-8.5, AAV CSp-8.6, AAV CSp-8.7, AAV CSp-8.8, AAV CSp-8.9, AAV CSp-9, AAV.hu.48R3, AAV.VR-355, AAV3B, AAV4, AAV5, AAVF1/HSC1, AAVF11/HSC11, AAVF12/HSC12, AAVF13/HSC13, AAVF14/HSC14, AAVF15/HSC15, AAVF16/HSC16, AAVF17/HSC17, AAVF2/HSC2, AAVF3/HSC3, AAVF4/HSC4, AAVF5/HSC5, AAVF6/HSC6, AAVF7/HSC7, AAVF8/HSC8, AAVF9/HSC9, AAVDJ, AAVDJ-8 PHP.B, PHP.A, G2B-26, G2B-13, TH1.1-32 и/или TH1.1-35.
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