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 PDFInfo
- 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
- Authority
- CA
- Canada
- Prior art keywords
- nucleic acid
- sequence
- rna
- genome editing
- seq
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
Classifications
-
- 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)
- 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)
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 (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)
| 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)
| 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 |
-
2017
- 2017-12-05 CN CN201780082711.XA patent/CN110168084A/zh active Pending
- 2017-12-05 CA CA3046076A patent/CA3046076A1/en active Pending
- 2017-12-05 US US15/832,567 patent/US9963719B1/en active Active
- 2017-12-05 KR KR1020197019386A patent/KR102604903B1/ko active Active
- 2017-12-05 MX MX2019006475A patent/MX2019006475A/es unknown
- 2017-12-05 JP JP2019530105A patent/JP7272952B2/ja active Active
- 2017-12-05 AU AU2017373797A patent/AU2017373797B2/en active Active
- 2017-12-05 WO PCT/US2017/064720 patent/WO2018106693A1/en not_active Ceased
- 2017-12-05 EP EP17829373.4A patent/EP3548614A1/en active Pending
-
2018
- 2018-03-23 US US15/934,750 patent/US10006054B1/en active Active
- 2018-05-15 US US15/980,450 patent/US10494649B2/en active Active
-
2019
- 2019-06-03 MX MX2024002327A patent/MX2024002327A/es unknown
- 2019-10-29 US US16/666,982 patent/US11028411B2/en active Active
-
2021
- 2021-05-27 US US17/332,764 patent/US11692205B2/en active Active
-
2022
- 2022-12-26 JP JP2022208855A patent/JP2023052089A/ja not_active Withdrawn
-
2023
- 2023-05-12 US US18/316,880 patent/US12264331B2/en active Active
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 |
| AU2017373797B2 (en) | 2024-08-29 |
| US20200056208A1 (en) | 2020-02-20 |
| WO2018106693A1 (en) | 2018-06-14 |
| CN110168084A (zh) | 2019-08-23 |
| US20180201956A1 (en) | 2018-07-19 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12264331B2 (en) | Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA | |
| JP7610559B2 (ja) | 合成ガイド分子、それに関連する組成物および方法 | |
| US12359223B2 (en) | Systems and methods for modulating chromosomal rearrangements | |
| US20240417706A1 (en) | Systems and methods for the treatment of hemoglobinopathies | |
| US20200299661A1 (en) | Cpf1-related methods and compositions for gene editing | |
| US20200155606A1 (en) | Crispr/rna-guided nuclease systems and methods | |
| US20240167059A1 (en) | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites | |
| EP3701028B1 (en) | Systems and methods for treating hyper-igm syndrome | |
| US20200263206A1 (en) | Targeted integration systems and methods for the treatment of hemoglobinopathies | |
| US20260083861A1 (en) | Crispr-related methods and compositions targeting cd70 expression | |
| NZ795268A (en) | Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA | |
| NZ795956A (en) | Synthetic guide molecules, compositions and methods relating thereto |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20220921 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D00-D120 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: VOLUNTARY SUBMISSION OF PRIOR ART RECEIVED Effective date: 20240905 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD Year of fee payment: 7 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20241202 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20241202 Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20241202 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250415 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D00-D123 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIOR ART DISCLOSURE DETERMINED COMPLIANT Effective date: 20250429 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20250429 |
|
| D15 | Examination report completed |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT Effective date: 20250724 |
|
| B12 | Application deemed to be withdrawn, abandoned or lapsed |
Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION Effective date: 20251124 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260116 |