CN115956120A - 用于crispr基因组编辑的修饰的指导rna - Google Patents

用于crispr基因组编辑的修饰的指导rna Download PDF

Info

Publication number
CN115956120A
CN115956120A CN202180047016.6A CN202180047016A CN115956120A CN 115956120 A CN115956120 A CN 115956120A CN 202180047016 A CN202180047016 A CN 202180047016A CN 115956120 A CN115956120 A CN 115956120A
Authority
CN
China
Prior art keywords
crrna
chemically modified
guide rna
tracrrna
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202180047016.6A
Other languages
English (en)
Chinese (zh)
Inventor
埃里克·约瑟夫·森特海默
阿纳斯塔西娅·科沃罗娃
乔纳森·肯尼斯·沃斯
纳迪亚·阿姆拉尼
陈泽祥
马修·哈斯勒
迪马斯·埃切维里亚·莫伦诺
朱莉娅·弗朗西斯·奥尔特曼
斯科特·沃尔夫
山田健
吉塔利·黛妃
张汉
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.)
University of Massachusetts Amherst
Original Assignee
University of Massachusetts Amherst
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of CN115956120A publication Critical patent/CN115956120A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/11DNA 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
    • 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
    • C12N15/87Introduction 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
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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]
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202180047016.6A 2020-05-12 2021-05-12 用于crispr基因组编辑的修饰的指导rna Pending CN115956120A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063023313P 2020-05-12 2020-05-12
US63/023,313 2020-05-12
PCT/US2021/032038 WO2021231606A2 (en) 2020-05-12 2021-05-12 Modified guide rnas for crispr genome editing

Publications (1)

Publication Number Publication Date
CN115956120A true CN115956120A (zh) 2023-04-11

Family

ID=78524997

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180047016.6A Pending CN115956120A (zh) 2020-05-12 2021-05-12 用于crispr基因组编辑的修饰的指导rna

Country Status (7)

Country Link
US (1) US20210388348A1 (https=)
EP (1) EP4150091A4 (https=)
JP (1) JP2023526057A (https=)
CN (1) CN115956120A (https=)
AU (1) AU2021271004A1 (https=)
CA (1) CA3177463A1 (https=)
WO (1) WO2021231606A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024053964A1 (ko) * 2022-09-06 2024-03-14 주식회사 툴젠 캄필로박터 제주니 유래 cas9의 가이드 rna 구조변화를 통한 유전자교정 향상 시스템
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015138510A1 (en) * 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
CN112877327B (zh) * 2014-12-18 2024-11-22 综合基因技术公司 基于crispr的组合物和使用方法
EP3250691B9 (en) * 2015-01-28 2023-08-02 Caribou Biosciences, Inc. Crispr hybrid dna/rna polynucleotides and methods of use
CN107709555A (zh) * 2015-05-15 2018-02-16 达尔马科恩有限公司 用于Cas9介导的基因编辑的合成的单向导RNA
EP4545544A3 (en) * 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017058751A1 (en) * 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
CA2947904A1 (en) * 2015-11-12 2017-05-12 Pfizer Inc. Tissue-specific genome engineering using crispr-cas9
CA3034931A1 (en) * 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
AU2017374044B2 (en) * 2016-12-08 2023-11-30 Intellia Therapeutics, Inc. Modified guide RNAs
WO2018111946A1 (en) * 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing detection
WO2019011118A1 (zh) * 2017-07-14 2019-01-17 苏州克睿基因生物科技有限公司 一种基因编辑系统及基因编辑的方法
WO2019183000A1 (en) * 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
KR20210045360A (ko) * 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019237069A1 (en) * 2018-06-08 2019-12-12 Intellia Therapeutics, Inc. Modified guide rnas for gene editing

Also Published As

Publication number Publication date
WO2021231606A3 (en) 2021-12-16
JP2023526057A (ja) 2023-06-20
EP4150091A4 (en) 2025-07-23
CA3177463A1 (en) 2021-11-18
WO2021231606A2 (en) 2021-11-18
AU2021271004A1 (en) 2023-01-19
US20210388348A1 (en) 2021-12-16
EP4150091A2 (en) 2023-03-22

Similar Documents

Publication Publication Date Title
US20210363518A1 (en) Modified guide rnas for crispr genome editing
AU2021282533C1 (en) Optimized crispr/cas9 systems and methods for gene editing in stem cells
EP3443088B1 (en) Grna fusion molecules, gene editing systems, and methods of use thereof
CN107690480B (zh) Cas9分子/指导rna分子复合物的评价
GB2617658A (en) Class II, type V CRISPR systems
CN110291198A (zh) 经修饰的指导rna
KR20010071227A (ko) 무세포 키메라플라스티 및 이형이중나선 돌연변이성벡터를 진핵세포에 이용
EP3781677A1 (en) Compositions and methods for improved gene editing
US20240218339A1 (en) Class ii, type v crispr systems
CN115956120A (zh) 用于crispr基因组编辑的修饰的指导rna
US20230183687A1 (en) Modified guide rnas for neisseria meningitidis cas9
US20240309396A1 (en) Compositions and methods for improved gene editing
US20240167029A1 (en) Modified guide rnas for crispr genome editing
US12612619B2 (en) Compositions and methods for improved gene editing
JP2025514327A (ja) Crisprガイドrnaのための保護オリゴヌクレオチド
Copp Repair of Intrastrand Alkylene Cross-links in Various DNA Structures by O6-Alkylguanine DNA Alkyltransferases
WO2024220306A1 (en) Chemically modified single stranded dna improves gene editing efficiency
EP4619534A2 (en) Effector proteins, compositions, systems and methods of use thereof
HK40014268A (en) Modified guide rnas

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
CB03 Change of inventor or designer information
CB03 Change of inventor or designer information

Inventor after: Eric Joseph Sentenheimer

Inventor after: Shan Tianjian

Inventor after: Guitari Daifei

Inventor after: Zhang Han

Inventor after: Amir mill

Inventor after: Anastasia Kovorova

Inventor after: Jonathan Kenneth Worth

Inventor after: Nadia Amrani

Inventor after: Chen Zexiang

Inventor after: Hassler Matthew

Inventor after: Dimas Echeveria Moreno

Inventor after: Julia Francis Altman

Inventor after: Scott Wolf

Inventor before: Eric Joseph Sentenheimer

Inventor before: Shan Tianjian

Inventor before: Guitari Daifei

Inventor before: Zhang Han

Inventor before: Anastasia Kovorova

Inventor before: Jonathan Kenneth Worth

Inventor before: Nadia Amrani

Inventor before: Chen Zexiang

Inventor before: Hassler Matthew

Inventor before: Dimas Echeveria Moreno

Inventor before: Julia Francis Altman

Inventor before: Scott Wolf