CA3046076A1 - Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna - Google Patents

Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna Download PDF

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Publication number
CA3046076A1
CA3046076A1 CA3046076A CA3046076A CA3046076A1 CA 3046076 A1 CA3046076 A1 CA 3046076A1 CA 3046076 A CA3046076 A CA 3046076A CA 3046076 A CA3046076 A CA 3046076A CA 3046076 A1 CA3046076 A1 CA 3046076A1
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Prior art keywords
nucleic acid
sequence
rna
genome editing
seq
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Pending
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CA3046076A
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English (en)
French (fr)
Inventor
Ari E. FRIEDLAND
Hariharan JAYARAM
Barrett Ethan Steinberg
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Editas Medicine Inc
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Editas Medicine Inc
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87Introduction 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
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    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/10Vectors comprising a non-peptidic targeting moiety

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3046076A 2016-12-05 2017-12-05 Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna Pending CA3046076A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662430154P 2016-12-05 2016-12-05
US62/430,154 2016-12-05
US201762503640P 2017-05-09 2017-05-09
US62/503,640 2017-05-09
PCT/US2017/064720 WO2018106693A1 (en) 2016-12-05 2017-12-05 SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA

Publications (1)

Publication Number Publication Date
CA3046076A1 true CA3046076A1 (en) 2018-06-14

Family

ID=60972337

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3046076A Pending CA3046076A1 (en) 2016-12-05 2017-12-05 Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna

Country Status (9)

Country Link
US (6) US9963719B1 (enExample)
EP (1) EP3548614A1 (enExample)
JP (2) JP7272952B2 (enExample)
KR (1) KR102604903B1 (enExample)
CN (1) CN110168084A (enExample)
AU (1) AU2017373797B2 (enExample)
CA (1) CA3046076A1 (enExample)
MX (2) MX2019006475A (enExample)
WO (1) WO2018106693A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
CA3001711A1 (en) * 2015-10-30 2017-05-04 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
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
US20190153440A1 (en) 2017-11-21 2019-05-23 Casebia Therapeutics Llp Materials and methods for treatment of autosomal dominant retinitis pigmentosa
WO2019118875A1 (en) * 2017-12-15 2019-06-20 Ou Li Crispr-mediated genome editing with vectors
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
WO2020055748A1 (en) * 2018-09-10 2020-03-19 The Regents Of The University Of California Modulators of cas9 polypeptides and methods of use thereof
WO2020176552A1 (en) 2019-02-25 2020-09-03 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2020186059A2 (en) * 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
WO2020264254A1 (en) * 2019-06-28 2020-12-30 Crispr Therapeutics Ag Materials and methods for controlling gene editing
WO2021053582A1 (en) * 2019-09-18 2021-03-25 Crispr Therapeutics Ag All-in-one self inactivating crispr vectors
WO2021062201A1 (en) * 2019-09-25 2021-04-01 Spotlight Therapeutics Compositions and methods for nucleoprotein targeting and expression
US20230203220A1 (en) * 2020-05-29 2023-06-29 Regents Of The University Of Minnesota Copolymers for intracellular therapeutic nucleic acid payload delivery
CA3200815A1 (en) * 2020-12-03 2022-06-09 Benjamin OAKES Compositions and methods for the targeting of bcl11a
WO2022221741A1 (en) 2021-04-16 2022-10-20 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2025179057A1 (en) * 2024-02-21 2025-08-28 The Regents Of The University Of California In vivo modification of cell genomes

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
SG10201804973TA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
KR102598856B1 (ko) * 2015-03-03 2023-11-07 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
WO2016176404A1 (en) * 2015-04-30 2016-11-03 The Brigham And Women's Hospital, Inc. Methods and kits for cloning-free genome editing
US20180291372A1 (en) * 2015-05-14 2018-10-11 Massachusetts Institute Of Technology Self-targeting genome editing system
CN107849547B (zh) 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
US11530421B2 (en) 2016-02-01 2022-12-20 The Regents Of The University Of California Self-inactivating endonuclease-encoding nucleic acids and methods of using the same
WO2017173004A1 (en) * 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing

Also Published As

Publication number Publication date
AU2017373797B2 (en) 2024-08-29
US20180201956A1 (en) 2018-07-19
US12264331B2 (en) 2025-04-01
US9963719B1 (en) 2018-05-08
US20220017927A1 (en) 2022-01-20
US10006054B1 (en) 2018-06-26
US11028411B2 (en) 2021-06-08
US10494649B2 (en) 2019-12-03
US20200056208A1 (en) 2020-02-20
KR20190088555A (ko) 2019-07-26
JP7272952B2 (ja) 2023-05-12
US20180251792A1 (en) 2018-09-06
JP2023052089A (ja) 2023-04-11
MX2024002327A (es) 2024-03-07
US11692205B2 (en) 2023-07-04
EP3548614A1 (en) 2019-10-09
MX2019006475A (es) 2019-09-26
CN110168084A (zh) 2019-08-23
KR102604903B1 (ko) 2023-11-21
JP2019536462A (ja) 2019-12-19
WO2018106693A1 (en) 2018-06-14
AU2017373797A1 (en) 2019-06-20
US20230416787A1 (en) 2023-12-28

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