AU2017373797B2 - 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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AU2017373797B2
AU2017373797B2 AU2017373797A AU2017373797A AU2017373797B2 AU 2017373797 B2 AU2017373797 B2 AU 2017373797B2 AU 2017373797 A AU2017373797 A AU 2017373797A AU 2017373797 A AU2017373797 A AU 2017373797A AU 2017373797 B2 AU2017373797 B2 AU 2017373797B2
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AU2017373797A1 (en
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Ari E. FRIEDLAND
Hariharan JAYARAM
Berrett Ethan Steinberg
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Editas Medicine Inc
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Editas Medicine Inc
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    • C12N9/14—Hydrolases (3)
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    • 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
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    • 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
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    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • 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
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/10—Vectors comprising a non-peptidic targeting moiety

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • 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)
AU2017373797A 2016-12-05 2017-12-05 Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA Active AU2017373797B2 (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 (2)

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AU2017373797A1 AU2017373797A1 (en) 2019-06-20
AU2017373797B2 true AU2017373797B2 (en) 2024-08-29

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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
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
CN109312338B (zh) * 2015-10-30 2022-09-27 爱迪塔斯医药公司 治疗单纯疱疹病毒的crispr/cas相关方法及组合物
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
EP3714055A1 (en) 2017-11-21 2020-09-30 CRISPR Therapeutics AG 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
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12227547B2 (en) 2018-09-10 2025-02-18 The Regents Of The University Of California Modulators of CAS9 polypeptides and methods of use thereof
US20220133768A1 (en) 2019-02-25 2022-05-05 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
US20220145274A1 (en) * 2019-03-12 2022-05-12 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
US12173290B2 (en) 2019-06-28 2024-12-24 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
EP4157903A2 (en) * 2020-05-29 2023-04-05 Regents of the University of Minnesota Copolymers for intracellular therapeutic nucleic acid payload delivery
EP4255502A4 (en) * 2020-12-03 2025-07-16 Scribe Therapeutics Inc COMPOSITIONS AND METHODS FOR TARGETING 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
CN111218447B (zh) * 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
KR20160097338A (ko) * 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
AU2016226077B2 (en) * 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
US20190002920A1 (en) * 2015-04-30 2019-01-03 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
PL3298134T3 (pl) 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
WO2017136335A1 (en) * 2016-02-01 2017-08-10 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

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Publication number Publication date
US10006054B1 (en) 2018-06-26
US20230416787A1 (en) 2023-12-28
EP3548614A1 (en) 2019-10-09
US9963719B1 (en) 2018-05-08
CN110168084A (zh) 2019-08-23
MX2019006475A (es) 2019-09-26
US11692205B2 (en) 2023-07-04
KR102604903B1 (ko) 2023-11-21
KR20190088555A (ko) 2019-07-26
US11028411B2 (en) 2021-06-08
CA3046076A1 (en) 2018-06-14
JP7272952B2 (ja) 2023-05-12
US10494649B2 (en) 2019-12-03
US20180251792A1 (en) 2018-09-06
US20220017927A1 (en) 2022-01-20
JP2019536462A (ja) 2019-12-19
MX2024002327A (es) 2024-03-07
US20180201956A1 (en) 2018-07-19
JP2023052089A (ja) 2023-04-11
US12264331B2 (en) 2025-04-01
US20200056208A1 (en) 2020-02-20
WO2018106693A1 (en) 2018-06-14
AU2017373797A1 (en) 2019-06-20

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