RU2015129018A - Рнк-направляемая инженерия генома человека - Google Patents

Рнк-направляемая инженерия генома человека Download PDF

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RU2015129018A
RU2015129018A RU2015129018A RU2015129018A RU2015129018A RU 2015129018 A RU2015129018 A RU 2015129018A RU 2015129018 A RU2015129018 A RU 2015129018A RU 2015129018 A RU2015129018 A RU 2015129018A RU 2015129018 A RU2015129018 A RU 2015129018A
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rna
cell
genomic dna
enzyme
eukaryotic cell
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Джордж М. ЧЁРЧ
Прашант МАЛИ
Лухан ЯНГ
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Президент Энд Фэллоуз Оф Харвард Коллидж
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Claims (26)

1. Способ изменения эукариотической клетки, предусматривающий
трансфекцию эукариотической клетки нуклеиновой кислотой, кодирующей РНК, комплементарную геномной ДНК этой эукариотической клетки,
трансфекцию эукариотической клетки нуклеиновой кислотой, кодирующей фермент, который взаимодействует с этой РНК и расщепляет геномную ДНК сайт-специфическим образом,
где эта клетка экспрессирует эти РНК и фермент,
РНК связывается с комплементарной геномной ДНК, а
фермент расщепляет геномную ДНК сайт-специфическим образом.
2. Способ по п. 1, где ферментом является Cas9.
3. Способ по п. 1, где эукариотическая клетка является клеткой дрожжей, клеткой растения или клеткой млекопитающего.
4. Способ по п. 1, где РНК содержит между приблизительно 10 - приблизительно 250 нуклеотидов.
5. Способ по п. 1, где РНК содержит между приблизительно 20 - приблизительно 100 нуклеотидов.
6. Способ изменения клетки человека, предусматривающий
трансфекцию клетки человека нуклеиновой кислотой, кодирующей РНК, комплементарную геномной ДНК эукариотической клетки,
трансфекцию клетки человека нуклеиновой кислотой, кодирующей фермент, который взаимодействует с РНК и расщепляет геномную ДНК сайт-специфическим образом,
где эта клетка человека экспрессирует эти РНК, и фермент, где РНК связывается с комплементарной геномной ДНК а фермент расщепляет геномную ДНК сайт-специфическим образом.
7. Способ по п. 6, где ферментом является Cas9.
8. Способ по п. 6, где РНК содержит между приблизительно 10 - приблизительно 250 нуклеотидов.
9. Способ по п. 6, где РНК содержит между приблизительно 20 - приблизительно 100 нуклеотидов.
10. Способ изменения эукариотической клетки во множестве сайтов геномной ДНК, предусматривающий
трансфекцию эукариотической клетки множеством нуклеиновых кислот,
кодирующих РНК, комплементарные различным сайтам геномной ДНК данной эукариотической клетки,
трансфекцию эукариотической клетки нуклеиновой кислотой, кодирующей фермент, который взаимодействует с этой РНК и расщепляет эти геномные ДНК сайт-специфическим образом,
где клетка экспрессирует эти РНК и фермент, эти РНК связываются с комплементарной геномной ДНК, а фермент расщепляет геномную ДНК сайт-специфическим образом.
11. Способ по п. 10, где этим ферментом является Cas9.
12. Способ по п. 10, где этой эукариотической клеткой является клетка дрожжей, клетка растения или клетка млекопитающего.
13. Способ по п. 10, где эта РНК содержит между приблизительно 10 - приблизительно 250 нуклеотидов.
14. Способ по п. 10, где эта РНК содержит между приблизительно 20 - приблизительно 100 нуклеотидов.
RU2015129018A 2012-12-17 2013-12-16 Рнк-направляемая инженерия генома человека RU2699523C2 (ru)

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US201261738355P 2012-12-17 2012-12-17
US61/738,355 2012-12-17
US201361779169P 2013-03-13 2013-03-13
US61/779,169 2013-03-13
PCT/US2013/075317 WO2014099744A1 (en) 2012-12-17 2013-12-16 Rna-guided human genome engineering

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