CA3047330A1 - Modified crispr rna and uses thereof - Google Patents

Modified crispr rna and uses thereof Download PDF

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Publication number
CA3047330A1
CA3047330A1 CA3047330A CA3047330A CA3047330A1 CA 3047330 A1 CA3047330 A1 CA 3047330A1 CA 3047330 A CA3047330 A CA 3047330A CA 3047330 A CA3047330 A CA 3047330A CA 3047330 A1 CA3047330 A1 CA 3047330A1
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Canada
Prior art keywords
compound
modified
crrna
recognition portion
sugar moiety
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.)
Abandoned
Application number
CA3047330A
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English (en)
French (fr)
Inventor
Meghdad RAHDAR
Thazha P. Prakash
Eric E. Swayze
C. Frank Bennett
Moira A. McMahon
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.)
Ludwig Institute for Cancer Research Ltd
Ionis Pharmaceuticals Inc
Original Assignee
Ludwig Institute for Cancer Research Ltd
Ionis Pharmaceuticals Inc
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 Ludwig Institute for Cancer Research Ltd, Ionis Pharmaceuticals Inc filed Critical Ludwig Institute for Cancer Research Ltd
Publication of CA3047330A1 publication Critical patent/CA3047330A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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 [CRISPR]
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3047330A 2016-12-28 2017-12-28 Modified crispr rna and uses thereof Abandoned CA3047330A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662439828P 2016-12-28 2016-12-28
US62/439,828 2016-12-28
US201762491818P 2017-04-28 2017-04-28
US62/491,818 2017-04-28
US201762572361P 2017-10-13 2017-10-13
US62/572,361 2017-10-13
PCT/US2017/068642 WO2018125964A1 (en) 2016-12-28 2017-12-28 Modified crispr rna and uses thereof

Publications (1)

Publication Number Publication Date
CA3047330A1 true CA3047330A1 (en) 2018-07-05

Family

ID=62710685

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3047330A Abandoned CA3047330A1 (en) 2016-12-28 2017-12-28 Modified crispr rna and uses thereof

Country Status (6)

Country Link
US (1) US20190382751A1 (OSRAM)
EP (1) EP3562942A4 (OSRAM)
JP (1) JP2020503017A (OSRAM)
AU (1) AU2017388379A1 (OSRAM)
CA (1) CA3047330A1 (OSRAM)
WO (1) WO2018125964A1 (OSRAM)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CN114269912A (zh) * 2019-06-14 2022-04-01 阿伯生物技术公司 新型crispr dna靶向酶及系统
KR20230090337A (ko) * 2020-10-19 2023-06-21 카리부 바이오사이언시스 인코포레이티드 Crispr 유형 v 시스템을 위한 dna-함유 폴리뉴클레오티드 및 가이드, 및 그의 제조 및 사용 방법
AU2023254100A1 (en) * 2022-04-13 2024-09-19 Caribou Biosciences, Inc. Therapeutic applications of crispr type v systems
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
CN116732075B (zh) * 2023-06-09 2024-03-08 江南大学 一种生产2′-岩藻糖基乳糖的多层动态调控系统及其应用
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases
WO2025231432A1 (en) * 2024-05-03 2025-11-06 Caribou Biosciences, Inc. In vivo gene editing with crispr systems

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102210319B1 (ko) * 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
US20150376587A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
EP4400584A3 (en) * 2014-12-03 2024-10-16 Agilent Technologies, Inc. Guide rna with chemical modifications
KR102319192B1 (ko) * 2015-01-28 2021-10-28 카리부 바이오사이언시스 인코포레이티드 Crispr 하이브리드 dna/rna 폴리뉴클레오티드 및 사용 방법
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP4545544A3 (en) * 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US12065667B2 (en) * 2016-04-16 2024-08-20 Ohio State Innovation Foundation Modified Cpf1 MRNA, modified guide RNA, and uses thereof
CN106244591A (zh) * 2016-08-23 2016-12-21 苏州吉玛基因股份有限公司 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用
US11136567B2 (en) * 2016-11-22 2021-10-05 Integrated Dna Technologies, Inc. CRISPR/CPF1 systems and methods

Also Published As

Publication number Publication date
EP3562942A1 (en) 2019-11-06
WO2018125964A1 (en) 2018-07-05
JP2020503017A (ja) 2020-01-30
EP3562942A4 (en) 2020-12-09
US20190382751A1 (en) 2019-12-19
AU2017388379A1 (en) 2019-06-06

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Effective date: 20230629