RU2018146815A - Прямое перепрограммировние соматических клеток человека в направлении выбранной (предопределенной) дифференцированной клетки с использованием функционализированных наночпстиц - Google Patents

Прямое перепрограммировние соматических клеток человека в направлении выбранной (предопределенной) дифференцированной клетки с использованием функционализированных наночпстиц Download PDF

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RU2018146815A
RU2018146815A RU2018146815A RU2018146815A RU2018146815A RU 2018146815 A RU2018146815 A RU 2018146815A RU 2018146815 A RU2018146815 A RU 2018146815A RU 2018146815 A RU2018146815 A RU 2018146815A RU 2018146815 A RU2018146815 A RU 2018146815A
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Андраник Эндрю АПРИКЯН
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Стемдженикс, Инк.
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Claims (40)

1. Композиция для индукции дифференцировки соматической клетки в интересующий специализированный тип клеток, содержащая по меньшей мере одно средство, индуцирующее специализированный тип клеток, конъюгированное с центральной наночастицей.
2. Композиция по п. 1, где по меньшей мере одно средство, индуцирующее специализированный тип клеток, конъюгировано с центральной наночастицей через первую функционализированную группу на наночастице.
3. Композиция по п. 1, где специализированный тип клеток представляет собой кардиомиоцит-подобную клетку (iCM), гепатоцит, нейрон, бета-клетку, клетку-предшественника крови, миоцит, остеобласт или другой тип клеток.
4. Композиция по п. 1, где по меньшей мере одно средство, индуцирующее специализированный тип клеток, включает по меньшей мере одно из средств, указанных в таблице 1, или его функциональный домен.
5. Композиция по одному из пп. 1-4, где по меньшей мере одно средство, индуцирующее специализированный тип клеток, включает два, три, четыре, пять или более молекул, указанных в таблице 1, или их функциональный домен.
6. Композиция по одному из пп. 1-4, где по меньшей мере одно средство, индуцирующее специализированный тип клеток, включает средства, указанные в таблице 1, или их функциональный домен.
7. Композиция по одному из пп. 1-4, где по меньшей мере одно средство, индуцирующее специализированный тип клеток, включает один или несколько белков или молекул РНК, указанных в таблице 1, или их функциональные домены.
8. Композиция по одному из пп. 4-7, где специализированный тип клеток представляет собой кардиомиоцит-подобную клетку (iCM), и одно или несколько средств, индуцирующих специализированный тип клеток, выбраны из Gata4, MEF2C, TBX5, MESP1, Hand2, MYOCD, miR-1 и miR-133.
9. Композиция по одному из пп. 1-8, дополнительно содержащая проникающий пептид (CPP), конъюгированный с наночастицей через вторую функционализированную группу на наночастице.
10. Композиция по одному из пп. 1-9, где наночастица имеет диаметр менее приблизительно 100 нм.
11. Композиция по п. 10, где наночастица имеет диаметр менее приблизительно 75, 50, 40 или 30 нм.
12. Композиция по одному из пп. 1-11, где центральная наночастица содержит молекулы железа или золота.
13. Композиция по одному из пп. 1-12, где центральная наночастица содержит полимерные молекулы.
14. Композиция по одному из пп. 1-13, где наночастица содержит полимерное покрытие.
15. Композиция по одному из пп. 9-14, где наночастица содержит полимерное покрытие, и первая и/или вторая функциональные группы присоединены к полимерному покрытию.
16. Композиция по одному из пп. 2-15, дополнительно содержащая первую линкерную молекулу, связывающую первую функциональную группу и по меньшей мере одно средство, индуцирующее специализированный тип клеток, указанное в таблице 1.
17. Композиция по одному из пп. 9-16, дополнительно содержащая вторую линкерную молекулу, связывающую вторую функциональную группу и CPP.
18. Композиция по п. 17, где первая линкерная молекула имеет первую длину, где вторая линкерная молекула имеет вторую длину, и где вторая длина больше первой длины.
19. Композиция по одному из пп. 9-18, где CPP содержит по меньшей мере пять основных аминокислот.
20. Композиция по п. 19, где CPP содержит приблизительно 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15 или более основных аминокислот.
21. Композиция по одному из пп. 19 и 20, где CPP содержит 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15 или более смежных основных аминокислот.
22. Клетка, содержащая композицию по любому из пп. 1-21.
23. Клетка по п. 22, где клетку получают из соматической клетки.
24. Клетка по п. 23, где клетку получают из фибробласта.
25. Клетка по п. 22, где клетка представляет собой интересующий индуцированный специализированный тип клеток.
26. Клетка по п. 25, где интересующий индуцированный специализированный тип клеток представляет собой кардиомиоцит-подобную клетку (iCM), гепатоцит, нейрон, бета-клетку, клетку-предшественника крови, миоцит, остеобласт или другой тип клеток.
27. Клетка по любому из пп. 22-26, где клетка является клеткой человека.
28. Способ индукции дифференцировки соматической клетки в интересующий специализированный тип клеток, указанный в таблице 1, включающий приведение соматической клетки в контакт с композицией по любому из пп. 1-21.
29. Способ по п. 28, где интересующий индуцированный специализированный тип клеток представляет собой кардиомиоцит-подобную клетку (iCM), гепатоцит, нейрон, бета-клетку, клетку-предшественник крови, миоцит, остеобласт или другой тип клеток.
30. Способ по одному из пп. 28 и 29, где соматическая клетка является фибробластом.
31. Способ по одному из пп. 28-30, где соматическую клетку приводят в контакт in vitro в условиях культивирования, достаточных для дифференцировки соматической клетки.
32. Способ по одному из пп. 28-31, где соматическая клетка является клеткой человека.
33. Способ скрининга фармацевтической композиции-кандидата in vitro на активность в интересующем индуцированном специализированном типе клеток, включающий:
приведение индуцированной специализированной клетки в контакт с фармацевтической композицией-кандидатом; и
наблюдение за индуцированной специализированной клеткой на предмет признаков активности.
34. Способ по п. 33, где индуцированная специализированная клетка выбрана из одного из типов клеток, указанных в таблице 1.
35. Способ по одному из пп. 33 и 34, где индуцированная специализированная клетка представляет собой кардиомиоцит-подобную клетку (iCM), гепатоцит, нейрон, бета-клетку, клетку-предшественника крови, миоцит, остеобласт или другой тип клеток.
36. Способ по одному из пп. 33-35, дополнительно включающий индукцию образования специализированной клетки из соматической клетки.
37. Способ по одному из пп. 33-36, где специализированную клетку индуцируют способом по одному из пп. 28-32.
38. Способ по одному из пп. 36 и 37, где соматическую клетку получают из нормального индивидуума или индивидуума с конкретным патологическим состоянием, и признак активности является признаком активности фармацевтической композиции для лечения патологического состояния у индивидуума.
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