CA3177463A1 - Modified guide rnas for crispr genome editing - Google Patents

Modified guide rnas for crispr genome editing Download PDF

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Publication number
CA3177463A1
CA3177463A1 CA3177463A CA3177463A CA3177463A1 CA 3177463 A1 CA3177463 A1 CA 3177463A1 CA 3177463 A CA3177463 A CA 3177463A CA 3177463 A CA3177463 A CA 3177463A CA 3177463 A1 CA3177463 A1 CA 3177463A1
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Prior art keywords
crrna
tracrrna
chemically modified
guide rna
rna
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Pending
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CA3177463A
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English (en)
French (fr)
Inventor
Erik Joseph SONTHEIMER
Anastasia Khvorova
Jonathan Kenneth WATTS
Nadia Amrani
Zexiang Chen
Matthew Hassler
Dimas Echeverria MORENO
Julia Frances ALTERMAN
Scot WOLFE
Ken Yamada
Gitali DEVI
Han Zhang
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University of Massachusetts Amherst
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Individual
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Publication of CA3177463A1 publication Critical patent/CA3177463A1/en
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • 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]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • 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)
CA3177463A 2020-05-12 2021-05-12 Modified guide rnas for crispr genome editing Pending CA3177463A1 (en)

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
CA3177463A1 true CA3177463A1 (en) 2021-11-18

Family

ID=78524997

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3177463A Pending CA3177463A1 (en) 2020-05-12 2021-05-12 Modified guide rnas for crispr genome editing

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
WO2021231606A2 (en) 2021-11-18
AU2021271004A1 (en) 2023-01-19
CN115956120A (zh) 2023-04-11
US20210388348A1 (en) 2021-12-16
EP4150091A2 (en) 2023-03-22

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