CN116096877B - Ii类ii型crispr系统 - Google Patents

Ii类ii型crispr系统

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Publication number
CN116096877B
CN116096877B CN202180039435.5A CN202180039435A CN116096877B CN 116096877 B CN116096877 B CN 116096877B CN 202180039435 A CN202180039435 A CN 202180039435A CN 116096877 B CN116096877 B CN 116096877B
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China
Prior art keywords
sequence
endonuclease
engineered
seq
domain
Prior art date
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Active
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CN202180039435.5A
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English (en)
Chinese (zh)
Other versions
CN116096877A (zh
Inventor
布莱恩·托马斯
克利斯多佛·布朗
奥德拉·德沃托
克里斯蒂娜·布特弗尔德
利萨·亚历山大
丹妮拉·S·A·戈尔茨曼
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Macrogenomics
Macrogenomics Therapy Co
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Macrogenomics
Macrogenomics Therapy Co
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Publication of CN116096877A publication Critical patent/CN116096877A/zh
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Confectionery (AREA)
  • Saccharide Compounds (AREA)
  • Seasonings (AREA)
CN202180039435.5A 2020-03-31 2021-03-30 Ii类ii型crispr系统 Active CN116096877B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063003159P 2020-03-31 2020-03-31
US63/003,159 2020-03-31
US202063116149P 2020-11-19 2020-11-19
US63/116,149 2020-11-19
PCT/US2021/024945 WO2021202568A1 (en) 2020-03-31 2021-03-30 Class ii, type ii crispr systems

Publications (2)

Publication Number Publication Date
CN116096877A CN116096877A (zh) 2023-05-09
CN116096877B true CN116096877B (zh) 2026-04-28

Family

ID=77929138

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180039435.5A Active CN116096877B (zh) 2020-03-31 2021-03-30 Ii类ii型crispr系统

Country Status (11)

Country Link
US (2) US11946039B2 (enExample)
EP (2) EP4632066A3 (enExample)
JP (2) JP7546689B2 (enExample)
KR (2) KR20250051161A (enExample)
CN (1) CN116096877B (enExample)
AU (1) AU2021248800A1 (enExample)
CA (1) CA3177051A1 (enExample)
ES (1) ES3055163T3 (enExample)
GB (1) GB2608292B (enExample)
MX (1) MX2022012110A (enExample)
WO (1) WO2021202568A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CN113728097A (zh) * 2019-02-14 2021-11-30 宏基因组学知识产权技术有限责任公司 具有ruvc结构域的酶
US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
US20230340537A1 (en) * 2020-01-17 2023-10-26 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
CA3177051A1 (en) 2020-03-31 2021-10-07 Metagenomi, Inc. Class ii, type ii crispr systems
CA3192224A1 (en) * 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
CN119452085A (zh) 2021-08-27 2025-02-14 宏基因组学公司 具有ruvc结构域的酶
MX2024005505A (es) 2021-11-05 2024-05-23 Metagenomi Inc Enzimas de edicion de bases.
WO2023097262A1 (en) * 2021-11-24 2023-06-01 Metagenomi, Inc. Endonuclease systems
KR20240110603A (ko) * 2021-11-24 2024-07-15 메타지노미, 인크. 엔도뉴클레아제 시스템
ES2970263B2 (es) * 2022-10-21 2024-10-11 Univ Alicante PROTEINA ENDONUCLEASA Cas9 Y SISTEMA CRISPR-Cas ASOCIADO
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024206759A1 (en) * 2023-03-31 2024-10-03 Arbor Biotechnologies, Inc. Crispr nuclease polypeptides and gene editing systems comprising such
CN121241133A (zh) * 2023-03-31 2025-12-30 阿伯生物技术公司 Crispr核酸酶多肽和包含此类crispr核酸酶多肽的基因编辑系统
WO2024206825A2 (en) * 2023-03-31 2024-10-03 Aera Therapeutics, Inc. Modified iscb nucleases, modified guide rnas, and uses thereof
WO2024233366A2 (en) * 2023-05-05 2024-11-14 Aera Therapeutics, Inc. Modified cas9-iid nucleases and uses thereof
JP2026519501A (ja) * 2023-05-23 2026-06-16 メタゲノミ セラピューティクス,インク. エンドヌクレアーゼ系
WO2025007044A1 (en) * 2023-06-28 2025-01-02 The University Of Chicago Methods and compositions comprising iscb variants
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025054425A1 (en) * 2023-09-08 2025-03-13 Arbor Biotechnologies, Inc. Reverse transcription-mediated gene editing systems and uses thereof
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116867897A (zh) * 2020-09-11 2023-10-10 宏基因组学公司 碱基编辑酶

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
US10584358B2 (en) 2013-10-30 2020-03-10 North Carolina State University Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
MX2017014560A (es) 2015-05-15 2018-03-01 Pioneer Hi Bred Int Caracterizacion rapida de sistemas de endonucleasa cas, secuencias pam y elementos arn de guia.
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
US20160362667A1 (en) 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
EP3929287A3 (en) 2015-06-18 2022-04-13 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
EP3426780A1 (en) 2016-03-11 2019-01-16 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
WO2018035250A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CA3038982A1 (en) * 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
WO2018074979A1 (en) 2016-10-17 2018-04-26 Nanyang Technological University Truncated crispr-cas proteins for dna targeting
WO2018209712A1 (en) 2017-05-19 2018-11-22 Tsinghua University Engineering of a minimal sacas9 crispr/cas system for gene editing and transcriptional regulation optimized by enhanced guide rna
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
CN110892069B (zh) 2017-03-30 2024-02-09 国立大学法人京都大学 基于基因组编辑的外显子跳跃诱导方法
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
WO2019161290A1 (en) 2018-02-15 2019-08-22 Sigma-Aldrich Co. Llc Engineered cas9 systems for eukaryotic genome modification
KR20200124702A (ko) 2018-02-23 2020-11-03 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 신규한 cas9 오르소로그
WO2020055941A1 (en) 2018-09-13 2020-03-19 Excision Biotherapeutics, Inc. Compositions and methods for excision with single grna
US20220220460A1 (en) 2019-02-14 2022-07-14 Metagenomi Ip Technologies, Llc Enzymes with ruvc domains
CN113728097A (zh) 2019-02-14 2021-11-30 宏基因组学知识产权技术有限责任公司 具有ruvc结构域的酶
US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
KR20240007322A (ko) * 2019-02-14 2024-01-16 메타지노미, 인크. Ruvc 도메인이 존재하는 효소
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
US20220403357A1 (en) * 2019-11-12 2022-12-22 The Broad Institute, Inc. Small type ii cas proteins and methods of use thereof
WO2021202559A1 (en) * 2020-03-31 2021-10-07 Metagenomi Ip Technologies, Llc Class ii, type ii crispr systems
CA3177051A1 (en) 2020-03-31 2021-10-07 Metagenomi, Inc. Class ii, type ii crispr systems
WO2021226369A1 (en) 2020-05-08 2021-11-11 Metagenomi Ip Technologies, Llc Enzymes with ruvc domains
CN116096892A (zh) 2020-05-08 2023-05-09 宏基因组学公司 具有RuvC结构域的酶
CA3192224A1 (en) * 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
EP4203993A4 (en) 2020-10-23 2024-10-23 The Broad Institute, Inc. Reprogrammable iscb nucleases and uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116867897A (zh) * 2020-09-11 2023-10-10 宏基因组学公司 碱基编辑酶

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Publication number Publication date
US11946039B2 (en) 2024-04-02
AU2021248800A1 (en) 2022-10-13
WO2021202568A1 (en) 2021-10-07
GB2608292A (en) 2022-12-28
JP7546689B2 (ja) 2024-09-06
US20240309356A1 (en) 2024-09-19
CN116096877A (zh) 2023-05-09
EP4127156A4 (en) 2024-03-27
JP7785139B2 (ja) 2025-12-12
CA3177051A1 (en) 2021-10-07
KR102794727B1 (ko) 2025-04-11
KR20250051161A (ko) 2025-04-16
EP4127156C0 (en) 2025-09-10
JP2024178181A (ja) 2024-12-24
EP4632066A3 (en) 2025-12-24
KR20220161383A (ko) 2022-12-06
MX2022012110A (es) 2022-10-18
GB202211839D0 (en) 2022-09-28
US20230051396A1 (en) 2023-02-16
EP4632066A2 (en) 2025-10-15
ES3055163T3 (en) 2026-02-10
EP4127156B1 (en) 2025-09-10
JP2023519953A (ja) 2023-05-15
GB2608292B (en) 2024-02-14
EP4127156A1 (en) 2023-02-08

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