MX2019006475A - Sistemas y métodos para dirección con arn guía puntual (arngp) de adn endógeno y fuente. - Google Patents

Sistemas y métodos para dirección con arn guía puntual (arngp) de adn endógeno y fuente.

Info

Publication number
MX2019006475A
MX2019006475A MX2019006475A MX2019006475A MX2019006475A MX 2019006475 A MX2019006475 A MX 2019006475A MX 2019006475 A MX2019006475 A MX 2019006475A MX 2019006475 A MX2019006475 A MX 2019006475A MX 2019006475 A MX2019006475 A MX 2019006475A
Authority
MX
Mexico
Prior art keywords
arngp
systems
methods
source
guide rna
Prior art date
Application number
MX2019006475A
Other languages
English (en)
Spanish (es)
Inventor
JAYARAM Hariharan
E Friedland Ari
Ethan Steinberg Barrett
Original Assignee
Editas Medicine 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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2019006475A publication Critical patent/MX2019006475A/es

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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
    • 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
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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]
    • 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
    • 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
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/10—Vectors comprising a non-peptidic targeting moiety

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (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)
MX2019006475A 2016-12-05 2017-12-05 Sistemas y métodos para dirección con arn guía puntual (arngp) de adn endógeno y fuente. MX2019006475A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662430154P 2016-12-05 2016-12-05
US201762503640P 2017-05-09 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
MX2019006475A true MX2019006475A (es) 2019-09-26

Family

ID=60972337

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019006475A MX2019006475A (es) 2016-12-05 2017-12-05 Sistemas y métodos para dirección con arn guía puntual (arngp) de adn endógeno y fuente.
MX2024002327A MX2024002327A (es) 2016-12-05 2019-06-03 Sistemas y metodos para direccion con arn guia puntual (arngp) de adn endogeno y fuente.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024002327A MX2024002327A (es) 2016-12-05 2019-06-03 Sistemas y metodos para direccion con arn guia puntual (arngp) de adn endogeno y fuente.

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
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

Also Published As

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
US11692205B2 (en) 2023-07-04
AU2017373797B2 (en) 2024-08-29
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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