JP7068821B2 - 化学修飾を有するガイドrna - Google Patents
化学修飾を有するガイドrna Download PDFInfo
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- JP7068821B2 JP7068821B2 JP2017529610A JP2017529610A JP7068821B2 JP 7068821 B2 JP7068821 B2 JP 7068821B2 JP 2017529610 A JP2017529610 A JP 2017529610A JP 2017529610 A JP2017529610 A JP 2017529610A JP 7068821 B2 JP7068821 B2 JP 7068821B2
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- Japan
- Prior art keywords
- certain embodiments
- guide rna
- nucleotide
- sequence
- synthetic
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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]
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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- C12N2310/00—Structure or type of the nucleic acid
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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]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/333—Modified A
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
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- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
- C12N2330/31—Libraries, arrays
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- Biochemistry (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022020615A JP7579820B2 (ja) | 2014-12-03 | 2022-02-14 | 化学修飾を有するガイドrna |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462087211P | 2014-12-03 | 2014-12-03 | |
| US62/087,211 | 2014-12-03 | ||
| US201562146189P | 2015-04-10 | 2015-04-10 | |
| US62/146,189 | 2015-04-10 | ||
| US201562256095P | 2015-11-16 | 2015-11-16 | |
| US62/256,095 | 2015-11-16 | ||
| PCT/US2015/000143 WO2016089433A1 (en) | 2014-12-03 | 2015-12-03 | Guide rna with chemical modifications |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022020615A Division JP7579820B2 (ja) | 2014-12-03 | 2022-02-14 | 化学修飾を有するガイドrna |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017537626A JP2017537626A (ja) | 2017-12-21 |
| JP2017537626A5 JP2017537626A5 (enExample) | 2019-01-17 |
| JP7068821B2 true JP7068821B2 (ja) | 2022-05-17 |
Family
ID=56092183
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017529610A Active JP7068821B2 (ja) | 2014-12-03 | 2015-12-03 | 化学修飾を有するガイドrna |
| JP2022020615A Active JP7579820B2 (ja) | 2014-12-03 | 2022-02-14 | 化学修飾を有するガイドrna |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022020615A Active JP7579820B2 (ja) | 2014-12-03 | 2022-02-14 | 化学修飾を有するガイドrna |
Country Status (8)
| Country | Link |
|---|---|
| US (5) | US10900034B2 (enExample) |
| EP (2) | EP4400584A3 (enExample) |
| JP (2) | JP7068821B2 (enExample) |
| KR (2) | KR102629128B1 (enExample) |
| CN (1) | CN107250148B (enExample) |
| AU (1) | AU2015355546B2 (enExample) |
| CA (1) | CA2969619A1 (enExample) |
| WO (1) | WO2016089433A1 (enExample) |
Families Citing this family (186)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| MX375420B (es) | 2014-02-11 | 2025-03-06 | Univ Colorado Regents | Ingenieria genetica multiplexada habilitada por crispr. |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CN107250148B (zh) | 2014-12-03 | 2021-04-16 | 安捷伦科技有限公司 | 具有化学修饰的指导rna |
| JP6718872B2 (ja) | 2014-12-17 | 2020-07-08 | プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップProQR Therapeutics II B.V. | 標的化rna編集 |
| JP2017538427A (ja) | 2014-12-18 | 2017-12-28 | インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド | Crispr系組成物及び使用方法 |
| BR112017017812A2 (pt) | 2015-02-23 | 2018-04-10 | Crispr Therapeutics Ag | materiais e métodos para tratamento de hemoglobinopatias |
| WO2016135559A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| CN114231527A (zh) | 2015-04-06 | 2022-03-25 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| AU2016285724A1 (en) * | 2015-06-29 | 2017-11-16 | Ionis Pharmaceuticals, Inc. | Modified CRISPR RNA and modified single CRISPR RNA and uses thereof |
| IL257105B (en) | 2015-07-31 | 2022-09-01 | Univ Minnesota | Modified cells and methods of therapy |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| EP3159407A1 (en) * | 2015-10-23 | 2017-04-26 | Silence Therapeutics (London) Ltd | Guide rnas, methods and uses |
| WO2017077394A2 (en) | 2015-11-04 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| WO2017136794A1 (en) * | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| US10767175B2 (en) * | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| JP7074345B2 (ja) | 2016-06-22 | 2022-05-24 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 一本鎖rna編集オリゴヌクレオチド |
| JP2019518478A (ja) | 2016-06-24 | 2019-07-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド,ア ボディー コーポレイトTHE REGENTS OF THE UNIVERSITY OF COLORADO,a body corporate | バーコードを付けたコンビナトリアルライブラリーを生成する方法 |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| EP3500671B1 (en) * | 2016-08-17 | 2024-07-10 | The Broad Institute, Inc. | Method of selecting target sequences for the design of guide rnas |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| IL264923B2 (en) | 2016-09-01 | 2025-07-01 | Proqr Therapeutics Ii Bv | Single-stranded RNA-editing oligonucleotides with chemical modifications |
| US11306305B2 (en) | 2016-09-23 | 2022-04-19 | Board Of Trustees Of Southern Illinois University | Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions |
| EP3516061A1 (en) | 2016-09-23 | 2019-07-31 | Synthena AG | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| WO2018055577A1 (en) * | 2016-09-23 | 2018-03-29 | Synthena Ag | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| US10669539B2 (en) * | 2016-10-06 | 2020-06-02 | Pioneer Biolabs, Llc | Methods and compositions for generating CRISPR guide RNA libraries |
| EP3523427A4 (en) | 2016-10-07 | 2020-09-02 | Integrated DNA Technologies, Inc. | S. PYOGENES AND POLYPEPTIDES MUTANT CAS9 GENES CODED BY SUCH GENES |
| US11242542B2 (en) | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
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| KR20170084334A (ko) | 2017-07-19 |
| EP3227447A1 (en) | 2017-10-11 |
| US20180051281A1 (en) | 2018-02-22 |
| CN107250148B (zh) | 2021-04-16 |
| US10337001B2 (en) | 2019-07-02 |
| CA2969619A1 (en) | 2016-06-09 |
| US20160289675A1 (en) | 2016-10-06 |
| JP7579820B2 (ja) | 2024-11-08 |
| EP3227447B1 (en) | 2024-04-24 |
| JP2017537626A (ja) | 2017-12-21 |
| US20250092389A1 (en) | 2025-03-20 |
| WO2016089433A1 (en) | 2016-06-09 |
| US10900034B2 (en) | 2021-01-26 |
| KR102629128B1 (ko) | 2024-01-25 |
| KR20240013283A (ko) | 2024-01-30 |
| US20210079389A1 (en) | 2021-03-18 |
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