AU2017388379A1 - Modified CRISPR RNA and uses thereof - Google Patents

Modified CRISPR RNA and uses thereof Download PDF

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Publication number
AU2017388379A1
AU2017388379A1 AU2017388379A AU2017388379A AU2017388379A1 AU 2017388379 A1 AU2017388379 A1 AU 2017388379A1 AU 2017388379 A AU2017388379 A AU 2017388379A AU 2017388379 A AU2017388379 A AU 2017388379A AU 2017388379 A1 AU2017388379 A1 AU 2017388379A1
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AU
Australia
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
AU2017388379A
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English (en)
Inventor
C. Frank Bennett
Moira A. McMahon
Thazha P. Prakash
Meghdad RAHDAR
Eric E. Swayze
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 AU2017388379A1 publication Critical patent/AU2017388379A1/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
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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]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (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)
AU2017388379A 2016-12-28 2017-12-28 Modified CRISPR RNA and uses thereof Abandoned AU2017388379A1 (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
AU2017388379A1 true AU2017388379A1 (en) 2019-06-06

Family

ID=62710685

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017388379A Abandoned AU2017388379A1 (en) 2016-12-28 2017-12-28 Modified CRISPR RNA and uses thereof

Country Status (6)

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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
AU2020291467A1 (en) * 2019-06-14 2021-12-23 Arbor Biotechnologies, Inc. Novel crispr DNA targeting enzymes and systems
WO2022086846A2 (en) * 2020-10-19 2022-04-28 Caribou Biosciences, Inc. Dna-containing polynucleotides and guides for crispr type v systems, and methods of making and using the same
US20250325698A1 (en) * 2022-04-13 2025-10-23 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
US9885033B2 (en) * 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
US20150376587A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
CN107250148B (zh) * 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
SG10201804715WA (en) * 2015-01-28 2018-07-30 Pioneer Hi Bred Int Crispr hybrid dna/rna polynucleotides and methods of use
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP3313989B1 (en) * 2015-06-29 2024-12-25 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017181107A2 (en) * 2016-04-16 2017-10-19 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
JP2019535287A (ja) * 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Crispr/cpf1システム及び方法

Also Published As

Publication number Publication date
US20190382751A1 (en) 2019-12-19
CA3047330A1 (en) 2018-07-05
JP2020503017A (ja) 2020-01-30
WO2018125964A1 (en) 2018-07-05
EP3562942A1 (en) 2019-11-06
EP3562942A4 (en) 2020-12-09

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