RU2015133046A - Двойной вектор для подавления вируса иммунодефицита человека - Google Patents

Двойной вектор для подавления вируса иммунодефицита человека Download PDF

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RU2015133046A
RU2015133046A RU2015133046A RU2015133046A RU2015133046A RU 2015133046 A RU2015133046 A RU 2015133046A RU 2015133046 A RU2015133046 A RU 2015133046A RU 2015133046 A RU2015133046 A RU 2015133046A RU 2015133046 A RU2015133046 A RU 2015133046A
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nucleic acid
hiv
promoter
vector
rna
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RU2015133046A
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Ирвин ЧЕН
Дон Сун ЭН
Мишель МИЛЛИНГТОН
Маурин БОЙД
Джеффри П. САЙМОНДС
Луис Рэндол БРЕТОН
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Кэлиммьюн Инк.
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Claims (25)

1. Способ получения вирусного экспрессионного вектора, который, присутствуя в клетке, может подавлять связывание ВИЧ с клеткой или предотвращать слияние ВИЧ с клеткой, который включает:
вставку первой молекулы нуклеиновой кислоты, кодирующей ингибирующую нуклеиновую кислоту, способную отрицательно регулировать экспрессию корецептора ВИЧ, в вектор; и
вставку второй молекулы нуклеиновой кислоты, кодирующей белок-ингибитор слияния ВИЧ, в вектор.
2. Способ по п. 1, в котором указанный вирусный экспрессионный вектор представляет собой лентивирусный вектор или ретровирусный вектор.
3. Способ по п. 2, в котором указанный лентивирусный вектор является самоинактивирующимся.
4. Способ по п. 2, в котором лентивирусный вектор представляет собой лентивирусный вектор FG11F.
5. Способ по любому из предшествующих пп., в котором белок-ингибитор слияния ВИЧ представляет собой белок С46, белок Т20, энфуфиртид, СР32М или сифувиртид.
6. Способ по п. 1, в котором ингибирующая нуклеиновая кислота представляет собой малую интерферирующую РНК (миРНК) или короткую шпилечную РНК (кшРНК), обладающую двухцепочечным участком.
7. Способ по п. 2, в котором ингибирующая нуклеиновая кислота является малой интерферирующей РНК (миРНК) или короткой шпилечной РНК (кшРНК), обладающая двухцепочечным участком.
8. Способ по п. 3, в котором ингибирующая нуклеиновая кислота является малой интерферирующей РНК (миРНК) или короткой шпилечной РНК (кшРНК), обладающая двухцепочечным участком.
9. Способ по п. 4, в котором ингибирующая нуклеиновая кислота является малой интерферирующей РНК (миРНК) или короткой шпилечной РНК (кшРНК), обладающей двухцепочечным участком.
10. Способ по п. 5, в котором ингибирующая нуклеиновая кислота является малой интерферирующей РНК (миРНК) или короткой шпилечной РНК (кшРНК), обладающей двухцепочечным участком.
11. Способ по п. 1, в котором указанный корецептор ВИЧ представляет собой CCR5 или CXCR4.
12. Способ по п. 2, в котором указанный корецептор ВИЧ представляет собой CCR5 или CXCR4.
13. Способ по п. 3, в котором указанный корецептор ВИЧ представляет собой CCR5 или CXCR4.
14. Способ по п. 4, в котором указанный корецептор ВИЧ представляет собой CCR5 или CXCR4.
15. Способ по п. 5, в котором указанный корецептор ВИЧ представляет собой CCR5 или CXCR4.
16. Способ по п. 11, в котором кшРНК имеет последовательность SEQ ID NO: 1.
17. Способ по п. 1, в котором белок-ингибитор слияния ВИЧ представляет собой белок С46 и тем, что ингибирующая нуклеиновая кислота представляет собой кшРНК, имеющую последовательность SEQ ID NO: 1.
18. Способ по п. 1, в котором первая последовательность нуклеиновых кислот функционально связана с первым промотором, а вторая последовательность нуклеиновых кислот функционально связана со вторым промотором.
19. Способ по п. 18, в котором указанный первый промотор является промотором РНК-полимеразы III, а указанный второй промотор является промотором РНК-полимеразы II.
20. Способ по п. 19, в котором промотор РНК-полимеразы III является промотором H1 полимеразы III.
21. Способ по п. 19, в котором промотор РНК-полимеразы II является промотором Убиквитин С полимеразы II.
22. Способ по п. 1, дополнительно включающий стадию вставки третьей молекулы нуклеиновой кислоты, которая кодирует ингибитор вирусной репликации ВИЧ в вектор.
23. Способ по п. 22, в котором ингибитор репликации ВИЧ выбирают из группы, содержащей: TRIM5α человека, TRIM5α макака-резус, химерный TRIM5α, белок слияния TRIM5 человека - циклофилин, циклофилин, Е3 убиквитин, APOBEC3G и антиген стромальных клеток костного мозга 2 (BST-2).
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