KR20230157387A - Cas 효소를 이용한 다중 편집 - Google Patents

Cas 효소를 이용한 다중 편집 Download PDF

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Publication number
KR20230157387A
KR20230157387A KR1020237034371A KR20237034371A KR20230157387A KR 20230157387 A KR20230157387 A KR 20230157387A KR 1020237034371 A KR1020237034371 A KR 1020237034371A KR 20237034371 A KR20237034371 A KR 20237034371A KR 20230157387 A KR20230157387 A KR 20230157387A
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South Korea
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sequence
rna
target
endonuclease
locus
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Pending
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KR1020237034371A
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English (en)
Korean (ko)
Inventor
브라이언 토마스
크리스토퍼 브라운
아우드라 데보토
크리스티나 버터필드
리사 알렉산더
다니엘라 에스 에이 골츠만
그레그 코스트
레베카 라모스
디에고 에스피노사
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메타지노미, 인크.
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Publication of KR20230157387A publication Critical patent/KR20230157387A/ko
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72Receptors; Cell surface antigens; Cell surface determinants for hormones
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Gastroenterology & Hepatology (AREA)
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  • Hematology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Graft Or Block Polymers (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
KR1020237034371A 2021-03-19 2022-03-18 Cas 효소를 이용한 다중 편집 Pending KR20230157387A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163163510P 2021-03-19 2021-03-19
US63/163,510 2021-03-19
US202163186506P 2021-05-10 2021-05-10
US63/186,506 2021-05-10
US202163241916P 2021-09-08 2021-09-08
US63/241,916 2021-09-08
PCT/US2022/021004 WO2022198080A1 (en) 2021-03-19 2022-03-18 Multiplex editing with cas enzymes

Publications (1)

Publication Number Publication Date
KR20230157387A true KR20230157387A (ko) 2023-11-16

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Family Applications (1)

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KR1020237034371A Pending KR20230157387A (ko) 2021-03-19 2022-03-18 Cas 효소를 이용한 다중 편집

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Country Link
US (1) US20250075233A1 (https=)
EP (1) EP4308699A4 (https=)
JP (1) JP2024515936A (https=)
KR (1) KR20230157387A (https=)
AU (1) AU2022237663A1 (https=)
BR (1) BR112023018948A2 (https=)
CA (1) CA3210361A1 (https=)
MX (1) MX2023010969A (https=)
WO (1) WO2022198080A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2617658B (en) 2020-03-06 2024-04-17 Metagenomi Inc Class II, type V CRISPR systems
KR20230021657A (ko) * 2020-05-08 2023-02-14 메타지노미, 인크. Ruvc 도메인을 포함하는 효소
BR112023014719A2 (pt) * 2021-01-22 2023-12-05 Metagenomi Inc Novas nucleases quiméricas e manipuladas
CA3225082A1 (en) 2021-08-27 2023-03-02 Brian C. Thomas Enzymes with ruvc domains
MX2024006257A (es) 2021-11-24 2024-07-29 Metagenomi Inc Sistemas de endonucleasas.
CA3277009A1 (en) * 2022-12-16 2024-06-20 Life Edit Therapeutics, Inc. RNA GUIDELINES TARGETING THE TRAC GENE AND METHODS OF USE

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WO2003099215A2 (en) * 2002-05-20 2003-12-04 Pharmacia Corporation Antisense modulation of glucocorticoid receptor expression
US20150017136A1 (en) * 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
US20180119174A1 (en) * 2015-05-13 2018-05-03 Seattle Children's Hospita (dba Seattle Children's Research Institute Enhancing endonuclease based gene editing in primary cells
KR102438360B1 (ko) * 2016-03-04 2022-08-31 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cpf1-관련 방법, 조성물 및 구성성분
MX2019013514A (es) * 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
TW201920661A (zh) * 2017-09-18 2019-06-01 大陸商博雅輯因(北京)生物科技有限公司 一種基因編輯t細胞及其用途
AU2019214935B2 (en) * 2018-01-30 2025-06-26 Editas Medicine, Inc. Systems and methods for modulating chromosomal rearrangements
US12275951B2 (en) * 2018-02-06 2025-04-15 Ohio State Innovation Foundation Engineered guide RNA and uses thereof
CA3089587A1 (en) * 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
US10913941B2 (en) * 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
AU2020223370B2 (en) * 2019-02-14 2023-04-20 Metagenomi Therapeutics, Inc. Enzymes with RuvC domains
US20220145333A1 (en) * 2019-03-11 2022-05-12 Sorrento Therapeutics, Inc. Improved process for integration of dna constructs using rna-guided endonucleases
GB201907733D0 (en) * 2019-05-31 2019-07-17 Governing Council Of The Univ Of Toronto Methods and compositions for multiplex gene editing

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Publication number Publication date
WO2022198080A1 (en) 2022-09-22
MX2023010969A (es) 2023-09-27
CA3210361A1 (en) 2022-09-22
AU2022237663A1 (en) 2023-10-12
US20250075233A1 (en) 2025-03-06
EP4308699A1 (en) 2024-01-24
EP4308699A4 (en) 2025-12-31
AU2022237663A9 (en) 2023-10-26
JP2024515936A (ja) 2024-04-11
BR112023018948A2 (pt) 2023-10-17

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