AU2021271004A1 - Modified guide RNAs for CRISPR genome editing - Google Patents

Modified guide RNAs for CRISPR genome editing Download PDF

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Publication number
AU2021271004A1
AU2021271004A1 AU2021271004A AU2021271004A AU2021271004A1 AU 2021271004 A1 AU2021271004 A1 AU 2021271004A1 AU 2021271004 A AU2021271004 A AU 2021271004A AU 2021271004 A AU2021271004 A AU 2021271004A AU 2021271004 A1 AU2021271004 A1 AU 2021271004A1
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AU
Australia
Prior art keywords
crrna
chemically modified
guide rna
tracrrna
rna
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Pending
Application number
AU2021271004A
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English (en)
Inventor
Julia Frances ALTERMAN
Nadia Amrani
Zexiang Chen
Gitali DEVI
Matthew Hassler
Anastasia Khvorova
Aamir MIR
Dimas Echeverria MORENO
Erik Joseph SONTHEIMER
Jonathan Kenneth WATTS
Scot WOLFE
Ken Yamada
Han Zhang
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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Publication date
Application filed by University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of AU2021271004A1 publication Critical patent/AU2021271004A1/en
Pending legal-status Critical Current

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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
    • C12N15/11—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
    • C12N15/87—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
    • 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
    • C12N2310/35—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)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021271004A 2020-05-12 2021-05-12 Modified guide RNAs for CRISPR genome editing Pending AU2021271004A1 (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
AU2021271004A1 true AU2021271004A1 (en) 2023-01-19

Family

ID=78524997

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021271004A Pending AU2021271004A1 (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
KR20240034661A (ko) * 2022-09-06 2024-03-14 주식회사 툴젠 캄필로박터 제주니 유래 Cas9의 가이드 RNA 구조변화를 통한 유전자교정 향상 시스템
AU2024270764A1 (en) 2023-05-15 2025-12-04 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
ES2745769T3 (es) * 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
CA2970683A1 (en) * 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
US9650617B2 (en) * 2015-01-28 2017-05-16 Pioneer Hi-Bred International. Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
JP2018515142A (ja) * 2015-05-15 2018-06-14 ダーマコン，インコーポレイテッド． Ｃａｓ９介在遺伝子編集用の合成シングルガイドｒｎａ
WO2017004261A1 (en) * 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
EP3356533A1 (en) * 2015-09-28 2018-08-08 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
SG10202106058WA (en) * 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
US20190032131A1 (en) * 2016-12-12 2019-01-31 Integrated Dna Technologies, Inc. Genome editing detection
US20210130817A1 (en) * 2017-07-14 2021-05-06 Cure Genetics Co., Ltd. Gene Editing System and Gene Editing Method
US20210363518A1 (en) * 2018-03-19 2021-11-25 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
AU2019282824C1 (en) * 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing

Also Published As

Publication number Publication date
US20210388348A1 (en) 2021-12-16
CN115956120A (zh) 2023-04-11
WO2021231606A3 (en) 2021-12-16
JP2023526057A (ja) 2023-06-20
EP4150091A4 (en) 2025-07-23
WO2021231606A2 (en) 2021-11-18
CA3177463A1 (en) 2021-11-18
EP4150091A2 (en) 2023-03-22

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Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE NAME OF THE INVENTOR TO READ SONTHEIMER, ERIK JOSEPH; KHVOROVA, ANASTASIA; WATTS, JONATHAN KENNETH; AMRANI, NADIA; CHEN, ZEXIANG; HASSLER, MATTHEW; MORENO, DIMAS ECHEVERRIA; ALTERMAN, JULIA FRANCES; WOLFE, SCOT; YAMADA, KEN; DEVI, GITALI; ZHANG, HAN AND MIR, AAMIR