RU2022103603A - Направляемые нуклеиновыми кислотами нуклеазы - Google Patents

Направляемые нуклеиновыми кислотами нуклеазы Download PDF

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RU2022103603A
RU2022103603A RU2022103603A RU2022103603A RU2022103603A RU 2022103603 A RU2022103603 A RU 2022103603A RU 2022103603 A RU2022103603 A RU 2022103603A RU 2022103603 A RU2022103603 A RU 2022103603A RU 2022103603 A RU2022103603 A RU 2022103603A
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nucleic acid
sequence
engineered
editing
seq
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RU2022103603A
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Джухан КИМ
Райан Т. ДЖИЛЛ
Эндрю ГАРСТ
Таня Элизабет Уорнеке ЛИПСКОМ
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Инскрипта, Инк.
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Priority claimed from US15/631,989 external-priority patent/US10011849B1/en
Priority claimed from US15/632,001 external-priority patent/US9982279B1/en
Application filed by Инскрипта, Инк. filed Critical Инскрипта, Инк.
Publication of RU2022103603A publication Critical patent/RU2022103603A/ru

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    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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Claims (8)

1. Способ модификации области-мишени в геноме клетки, включающий приведение клетки в контакт с направляемой нуклеиновой кислотой нуклеазой, содержащей:
(a) нуклеиновую кислоту, по меньшей мере на 95% идентичную последовательности нуклеиновой кислоты SEQ ID NO:24 или SEQ ID NO:44, где нуклеиновая кислота кодирует направляемую нуклеиновой кислотой нуклеазу, содержащую аминокислотную последовательность SEQ ID NO: 4;
(b) сконструированную направляющую нуклеиновую кислоту, способную образовывать комплекс с направляемой нуклеиновой кислотой нуклеазой, при этом последовательность сконструированной направляющей нуклеиновой кислоты сконструирована для гибридизации с последовательностью TTTN области-мишени в геноме клетки; и
(c) редактирующую последовательность, кодирующую нуклеиновую кислоту, комплементарную указанной области-мишени, имеющей изменение в последовательности, по сравнению с областью-мишенью;
где система приводит к геномному редактированию в области-мишени в геноме клетки, облегчаемому нуклеазой, сконструированной направляющей нуклеиновой кислотой и редактирующей последовательностью.
2. Способ по п.1, где сконструированная направляющая нуклеиновая кислота и редактирующая последовательность представлены в форме одиночной нуклеиновой кислоты.
3. Способ по п.1, где сконструированная направляющая нуклеиновая кислота содержит последовательность SEQ ID NO: 87.
4. Способ по п.1, где редактирующая последовательность содержит мутацию в участке мотива, смежного с протоспейсером (PAM).
RU2022103603A 2017-06-23 2018-05-25 Направляемые нуклеиновыми кислотами нуклеазы RU2022103603A (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US15/632,001 2017-06-23
US15/631,989 US10011849B1 (en) 2017-06-23 2017-06-23 Nucleic acid-guided nucleases
US15/632,001 US9982279B1 (en) 2017-06-23 2017-06-23 Nucleic acid-guided nucleases
US15/631,989 2017-06-23

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CN (1) CN111511906A (ru)
AU (2) AU2018289077B2 (ru)
CA (1) CA3067951A1 (ru)
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RU2020102451A3 (ru) 2021-11-25
CN111511906A (zh) 2020-08-07
IL271342A (en) 2020-01-30
JP2020530264A (ja) 2020-10-22
MX2019015047A (es) 2020-08-03
RU2769475C2 (ru) 2022-04-01
NZ760730A (en) 2023-04-28
KR102558931B1 (ko) 2023-07-21
RU2020102451A (ru) 2021-07-26
WO2018236548A1 (en) 2018-12-27
EP3642334A1 (en) 2020-04-29
JP7136816B2 (ja) 2022-09-13
AU2022202248A1 (en) 2022-04-21
KR20210132244A (ko) 2021-11-03
ES2971549T3 (es) 2024-06-05
KR20200020903A (ko) 2020-02-26
AU2022202248B2 (en) 2024-09-19
KR102321388B1 (ko) 2021-11-03
EP3916086A1 (en) 2021-12-01
EP3642334A4 (en) 2021-03-24
AU2018289077B2 (en) 2022-03-10
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