JP6625971B2 - 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 - Google Patents
配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 Download PDFInfo
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- JP6625971B2 JP6625971B2 JP2016521449A JP2016521449A JP6625971B2 JP 6625971 B2 JP6625971 B2 JP 6625971B2 JP 2016521449 A JP2016521449 A JP 2016521449A JP 2016521449 A JP2016521449 A JP 2016521449A JP 6625971 B2 JP6625971 B2 JP 6625971B2
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- C—CHEMISTRY; METALLURGY
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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]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
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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
- 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
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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
- 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]
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- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Bioinformatics & Computational Biology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361836123P | 2013-06-17 | 2013-06-17 | |
| US61/836,123 | 2013-06-17 | ||
| US201361847537P | 2013-07-17 | 2013-07-17 | |
| US61/847,537 | 2013-07-17 | ||
| US201361862468P | 2013-08-05 | 2013-08-05 | |
| US201361862355P | 2013-08-05 | 2013-08-05 | |
| US61/862,355 | 2013-08-05 | ||
| US61/862,468 | 2013-08-05 | ||
| US201361871301P | 2013-08-28 | 2013-08-28 | |
| US61/871,301 | 2013-08-28 | ||
| US201361915407P | 2013-12-12 | 2013-12-12 | |
| US61/915,407 | 2013-12-12 | ||
| US201461979942P | 2014-04-15 | 2014-04-15 | |
| US61/979,942 | 2014-04-15 | ||
| PCT/US2014/041800 WO2014204724A1 (en) | 2013-06-17 | 2014-06-10 | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019215361A Division JP2020054354A (ja) | 2013-06-17 | 2019-11-28 | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016521993A JP2016521993A (ja) | 2016-07-28 |
| JP2016521993A5 JP2016521993A5 (enExample) | 2019-10-03 |
| JP6625971B2 true JP6625971B2 (ja) | 2019-12-25 |
Family
ID=51059658
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016521449A Active JP6625971B2 (ja) | 2013-06-17 | 2014-06-10 | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
| JP2019215361A Pending JP2020054354A (ja) | 2013-06-17 | 2019-11-28 | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019215361A Pending JP2020054354A (ja) | 2013-06-17 | 2019-11-28 | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11008588B2 (enExample) |
| EP (2) | EP3674411A1 (enExample) |
| JP (2) | JP6625971B2 (enExample) |
| KR (1) | KR20160044457A (enExample) |
| CN (1) | CN106062197A (enExample) |
| AU (1) | AU2014281026B2 (enExample) |
| CA (1) | CA2915834A1 (enExample) |
| DK (1) | DK3011029T3 (enExample) |
| ES (1) | ES2777217T3 (enExample) |
| WO (1) | WO2014204724A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12447213B2 (en) | 2016-10-07 | 2025-10-21 | The Broad Institute, Inc. | Modulation of novel immune checkpoint targets |
Families Citing this family (306)
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| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| LT3401400T (lt) | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
| JP2015527889A (ja) * | 2012-07-25 | 2015-09-24 | ザ ブロード インスティテュート， インコーポレイテッド | 誘導可能なｄｎａ結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用 |
| PL4397760T3 (pl) | 2012-10-23 | 2026-03-30 | Toolgen Incorporated | Kompozycja do rozszczepiania docelowego dna zawierająca prowadzące rna specyficzne dla docelowego dna i kwas nukleinowy kodujący białko cas lub białko cas oraz jej zastosowanie |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| IL239317B (en) | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications |
| CN114634950A (zh) | 2012-12-12 | 2022-06-17 | 布罗德研究所有限公司 | 用于序列操纵的crispr-cas组分系统、方法以及组合物 |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| US10378027B2 (en) * | 2013-03-15 | 2019-08-13 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| DK2986729T3 (en) | 2013-04-16 | 2018-10-29 | Regeneron Pharma | TARGETED MODIFICATION OF ROOT THROUGH |
| MX2015017312A (es) | 2013-06-17 | 2017-04-10 | Broad Inst Inc | Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas. |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| JP6665088B2 (ja) | 2013-06-17 | 2020-03-13 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物 |
| CN105793425B (zh) * | 2013-06-17 | 2021-10-26 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
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| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
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| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
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| WO2015066119A1 (en) | 2013-10-30 | 2015-05-07 | North Carolina State University | Compositions and methods related to a type-ii crispr-cas system in lactobacillus buchneri |
| 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 |
| WO2015088643A1 (en) | 2013-12-11 | 2015-06-18 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a genome |
| CN111206032B (zh) * | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| RU2016128077A (ru) | 2013-12-12 | 2018-12-06 | Те Брод Инститьют Инк. | Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
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| BR112016013547A2 (pt) | 2013-12-12 | 2017-10-03 | Broad Inst Inc | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
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| KR102796744B1 (ko) | 2015-06-09 | 2025-04-15 | 에디타스 메디신, 인코포레이티드 | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12447213B2 (en) | 2016-10-07 | 2025-10-21 | The Broad Institute, Inc. | Modulation of novel immune checkpoint targets |
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| AU2014281026A9 (en) | 2016-06-16 |
| AU2014281026B2 (en) | 2020-05-28 |
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| JP2016521993A (ja) | 2016-07-28 |
| EP3674411A1 (en) | 2020-07-01 |
| WO2014204724A9 (en) | 2016-03-24 |
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