WO2019009682A3 - 표적 특이적 crispr 변이체 - Google Patents

표적 특이적 crispr 변이체 Download PDF

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Publication number
WO2019009682A3
WO2019009682A3 PCT/KR2018/007731 KR2018007731W WO2019009682A3 WO 2019009682 A3 WO2019009682 A3 WO 2019009682A3 KR 2018007731 W KR2018007731 W KR 2018007731W WO 2019009682 A3 WO2019009682 A3 WO 2019009682A3
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target
specific crispr
genome
artificially manipulated
crispr mutant
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PCT/KR2018/007731
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English (en)
French (fr)
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WO2019009682A2 (ko
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김영훈
정민희
이준선
신은지
이강인
김석중
이정준
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주식회사 툴젠
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Priority to CN201880058272.3A priority Critical patent/CN111094561B/zh
Priority to CA3069296A priority patent/CA3069296A1/en
Priority to BR112020000310-7A priority patent/BR112020000310A2/pt
Priority to SG11201913852RA priority patent/SG11201913852RA/en
Priority to EP18829020.9A priority patent/EP3650540A4/en
Priority to AU2018295992A priority patent/AU2018295992B2/en
Priority to JP2020500033A priority patent/JP2020530279A/ja
Priority to RU2020105703A priority patent/RU2020105703A/ru
Application filed by 주식회사 툴젠 filed Critical 주식회사 툴젠
Priority to US16/628,199 priority patent/US11441135B2/en
Publication of WO2019009682A2 publication Critical patent/WO2019009682A2/ko
Publication of WO2019009682A3 publication Critical patent/WO2019009682A3/ko
Priority to AU2022200262A priority patent/AU2022200262B2/en
Priority to US17/880,160 priority patent/US20230130858A1/en
Priority to JP2022181946A priority patent/JP2023025045A/ja

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 [CRISPRs]

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  • Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract

본 발명은 인위적으로 조작된 CRISPR/Cas9 시스템에 관한 것이다. 보다 구체적으로, 표적 특이성이 향상된 인위적으로 조작된 CRISPR 효소 및 이를 포함하는 인위적으로 조작된 CRISPR/Cas9 시스템을 이용한 게놈 및/또는 후생유전자(epigenome)의 조작 또는 변형, 게놈 표적화, 게놈 교정 및 체외 진단 등의 용도에 관한 것이다.
PCT/KR2018/007731 2017-07-07 2018-07-06 표적 특이적 crispr 변이체 WO2019009682A2 (ko)

Priority Applications (12)

Application Number Priority Date Filing Date Title
JP2020500033A JP2020530279A (ja) 2017-07-07 2018-07-06 標的特異的なcrisprバリアント
BR112020000310-7A BR112020000310A2 (pt) 2017-07-07 2018-07-06 variantes de crispr alvo específicas
SG11201913852RA SG11201913852RA (en) 2017-07-07 2018-07-06 Target-specific crispr mutant
EP18829020.9A EP3650540A4 (en) 2017-07-07 2018-07-06 TARGET-SPECIFIC CRISPR MUTANT
AU2018295992A AU2018295992B2 (en) 2017-07-07 2018-07-06 Target-specific CRISPR mutant
CN201880058272.3A CN111094561B (zh) 2017-07-07 2018-07-06 靶特异性crispr变体
RU2020105703A RU2020105703A (ru) 2017-07-07 2018-07-06 Специфические для мишени варианты, связанные с crispr
CA3069296A CA3069296A1 (en) 2017-07-07 2018-07-06 Target-specific crispr mutant
US16/628,199 US11441135B2 (en) 2017-07-07 2018-07-06 Target-specific CRISPR mutant
AU2022200262A AU2022200262B2 (en) 2017-07-07 2022-01-17 Target-specific CRISPR mutant
US17/880,160 US20230130858A1 (en) 2017-07-07 2022-08-03 Target-specific crispr mutant
JP2022181946A JP2023025045A (ja) 2017-07-07 2022-11-14 標的特異的なcrisprバリアント

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762529601P 2017-07-07 2017-07-07
US62/529,601 2017-07-07
US201762608722P 2017-12-21 2017-12-21
US62/608,722 2017-12-21

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US16/628,199 A-371-Of-International US11441135B2 (en) 2017-07-07 2018-07-06 Target-specific CRISPR mutant
US17/880,160 Continuation US20230130858A1 (en) 2017-07-07 2022-08-03 Target-specific crispr mutant

Publications (2)

Publication Number Publication Date
WO2019009682A2 WO2019009682A2 (ko) 2019-01-10
WO2019009682A3 true WO2019009682A3 (ko) 2019-03-28

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PCT/KR2018/007731 WO2019009682A2 (ko) 2017-07-07 2018-07-06 표적 특이적 crispr 변이체

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US (2) US11441135B2 (ko)
EP (1) EP3650540A4 (ko)
JP (2) JP2020530279A (ko)
KR (1) KR102691097B1 (ko)
CN (1) CN111094561B (ko)
AU (2) AU2018295992B2 (ko)
BR (1) BR112020000310A2 (ko)
CA (1) CA3069296A1 (ko)
RU (1) RU2020105703A (ko)
SG (1) SG11201913852RA (ko)
WO (1) WO2019009682A2 (ko)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018201086A1 (en) 2017-04-28 2018-11-01 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
US11441135B2 (en) * 2017-07-07 2022-09-13 Toolgen Incorporated Target-specific CRISPR mutant
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US11713452B2 (en) * 2017-09-08 2023-08-01 University Of North Texas Health Science Center Engineered CAS9 variants
KR20200125191A (ko) 2019-04-26 2020-11-04 주식회사 툴젠 표적 특이적 crispr 변이체
CN114026226A (zh) * 2019-04-26 2022-02-08 株式会社图尔金 靶特异性crispr突变体
EP4093863A4 (en) * 2020-01-24 2024-04-10 The General Hospital Corporation CRISPR-CAS ENZYMES WITH IMPROVED ON-TARGET ACTIVITY
WO2021171048A1 (en) * 2020-02-25 2021-09-02 Biospirál-2006 Fejlesztő És Tanácsadó Kft. Variant cas9
CN112430613A (zh) * 2020-12-08 2021-03-02 安徽省农业科学院水稻研究所 一种宽编辑范围的SpG基因及其应用
WO2022120439A1 (en) * 2020-12-11 2022-06-16 The University Of Western Australia Enzyme variants
CN112538471B (zh) * 2020-12-28 2023-12-12 南方医科大学 一种CRISPR SpCas9(K510A)突变体及其应用
KR102567576B1 (ko) * 2020-12-31 2023-08-14 한양대학교 산학협력단 표적 특이성이 향상된 신규한 Cas9 단백질 변이체 및 이의 용도

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016115355A1 (en) * 2015-01-14 2016-07-21 Temple University-Of The Commonwealth System Of Higher Education Rna guided eradication of herpes simplex type i and other related herpesviruses
US20160319260A1 (en) * 2015-03-03 2016-11-03 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with Altered PAM Specificity
WO2016205613A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
WO2017040348A1 (en) * 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
WO2018034554A1 (ko) * 2016-08-19 2018-02-22 주식회사 툴젠 인위적으로 조작된 신생혈관형성 조절 시스템

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ712727A (en) * 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
KR20160097327A (ko) * 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 cas 효소의 발현의 변경을 위한 crispr-cas 시스템 및 방법
EP3277805A1 (en) * 2015-03-31 2018-02-07 Exeligen Scientific, Inc. Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
US11008555B2 (en) 2015-09-17 2021-05-18 The Regents Of The University Of California Variant Cas9 polypeptides comprising internal insertions
JP6940117B2 (ja) 2016-06-15 2021-09-22 ツールゲン インコーポレイテッドToolgen Incorporated オンターゲットおよびオフターゲットの多標的システムを用いた標的特異的ヌクレアーゼをスクリーニングするための方法およびその利用
BR112019002779A2 (pt) 2016-08-12 2019-05-14 Toolgen Incorporated elemento imunorregulador manipulado e imunidade alterada por este
US11441135B2 (en) * 2017-07-07 2022-09-13 Toolgen Incorporated Target-specific CRISPR mutant

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016115355A1 (en) * 2015-01-14 2016-07-21 Temple University-Of The Commonwealth System Of Higher Education Rna guided eradication of herpes simplex type i and other related herpesviruses
US20160319260A1 (en) * 2015-03-03 2016-11-03 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with Altered PAM Specificity
WO2016205613A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
WO2017040348A1 (en) * 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
WO2018034554A1 (ko) * 2016-08-19 2018-02-22 주식회사 툴젠 인위적으로 조작된 신생혈관형성 조절 시스템

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DATABASE Protein 7 October 2015 (2015-10-07), "type II CRISPR RNA-guided endonuclease Cas9 [Streptococcus pyogenes]", XP055586425, retrieved from NCBI Database accession no. WP_010922251.1 *

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Publication number Publication date
EP3650540A4 (en) 2021-03-31
US20200149022A1 (en) 2020-05-14
EP3650540A2 (en) 2020-05-13
SG11201913852RA (en) 2020-01-30
AU2022200262A1 (en) 2022-02-10
CN111094561A (zh) 2020-05-01
BR112020000310A2 (pt) 2020-07-14
CA3069296A1 (en) 2019-01-10
US11441135B2 (en) 2022-09-13
CN111094561B (zh) 2024-03-12
AU2018295992A1 (en) 2020-01-30
WO2019009682A2 (ko) 2019-01-10
RU2020105703A (ru) 2021-08-09
KR102691097B1 (ko) 2024-08-02
AU2018295992B2 (en) 2021-10-21
KR20190005801A (ko) 2019-01-16
AU2022200262B2 (en) 2024-01-04
JP2020530279A (ja) 2020-10-22
JP2023025045A (ja) 2023-02-21
US20230130858A1 (en) 2023-04-27

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