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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nucleic acid
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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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    • C12N9/14Hydrolases (3)
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    • 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
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    • 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
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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]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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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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    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/10Vectors comprising a non-peptidic targeting moiety

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  • 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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AU2017373797A Active AU2017373797B2 (en) 2016-12-05 2017-12-05 Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA

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

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106459995B (zh) 2013-11-07 2020-02-21 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
CN115491373A (zh) * 2015-10-30 2022-12-20 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
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
CA3082450A1 (en) 2017-11-21 2019-05-31 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
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED 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
CA3130515A1 (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)
SG11202109741VA (en) * 2019-03-12 2021-10-28 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
MA56393A (fr) * 2019-06-28 2022-05-04 Bayer Healthcare Llc Matériels et méthodes de régulation de l'édition de gènes
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
CA3180775A1 (en) * 2020-05-29 2021-12-16 Regents Of The University Of Minnesota Copolymers for intracellular therapeutic nucleic acid payload delivery
AU2021391783A1 (en) * 2020-12-03 2023-06-22 Scribe Therapeutics Inc. 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
CN106459995B (zh) 2013-11-07 2020-02-21 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
JP6712948B2 (ja) * 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法
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)
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
KR20230156800A (ko) * 2015-03-03 2023-11-14 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
BR112017024514A2 (pt) 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
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

Also Published As

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

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