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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- C—CHEMISTRY; METALLURGY
- 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
- C12N15/11—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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- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- 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
- C12N2310/10—Type of 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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- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
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- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
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- Medicinal Chemistry (AREA)
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- Cell Biology (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) | EP3227447B1 (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 (187)
| 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 |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| DE212015000061U1 (de) | 2014-02-11 | 2017-09-03 | The Regents Of The University Of Colorado, A Body Corporate | CRISPR-ermöglichtes Multiplex Genom Engineering |
| CA2956224A1 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US10900034B2 (en) | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| CA2968336C (en) | 2014-12-17 | 2021-11-23 | Proqr Therapeutics Ii B.V. | Construct for site directed editing of an adenosine nucleotide in target rna |
| EP3786296A1 (en) | 2014-12-18 | 2021-03-03 | Integrated Dna Technologies, Inc. | Crispr-based compositions and methods of use |
| BR112017017810A2 (pt) | 2015-02-23 | 2018-04-10 | Crispr Therapeutics Ag | materiais e métodos para tratamento de hemoglobinopatias |
| US12129471B2 (en) | 2015-02-23 | 2024-10-29 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| KR102888521B1 (ko) | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| US11414657B2 (en) * | 2015-06-29 | 2022-08-16 | Ionis Pharmaceuticals, Inc. | Modified CRISPR RNA and modified single CRISPR RNA and uses thereof |
| US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| IL295616A (en) | 2015-07-31 | 2022-10-01 | Us Health | Adapted cells and treatment methods |
| 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 |
| EP4434589A3 (en) | 2015-10-23 | 2025-05-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| EP3159407A1 (en) * | 2015-10-23 | 2017-04-26 | Silence Therapeutics (London) Ltd | Guide rnas, methods and uses |
| ES2942309T3 (es) | 2015-11-04 | 2023-05-31 | Vertex Pharma | Materiales y métodos para el tratamiento de hemoglobinopatías |
| 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 |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| CN109688820B (zh) | 2016-06-24 | 2023-01-10 | 科罗拉多州立大学董事会(法人团体) | 用于生成条形码化组合文库的方法 |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018035388A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3507366B1 (en) | 2016-09-01 | 2020-10-07 | ProQR Therapeutics II B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| CA3034931A1 (en) | 2016-09-23 | 2018-03-29 | Board Of Trustees Of Southern Illinois University | Tuning crispr/cas9 activity with chemically modified nucleotide substitutions |
| US11118179B2 (en) | 2016-09-23 | 2021-09-14 | 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 |
| CN110462034A (zh) | 2016-10-07 | 2019-11-15 | 综合Dna技术公司 | 化脓链球菌cas9突变基因和由其编码的多肽 |
| US11242542B2 (en) | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| EP4338799A3 (en) | 2016-10-18 | 2024-06-05 | Regents of the University of Minnesota | Tumor infiltrating lymphocytes and methods of therapy |
| EP3315125A1 (en) | 2016-10-31 | 2018-05-02 | Silence Therapeutics (London) Ltd | Lipid nanoparticle formulation |
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| AU2015355546A1 (en) | 2017-07-13 |
| EP4400584A2 (en) | 2024-07-17 |
| JP7579820B2 (ja) | 2024-11-08 |
| KR102629128B1 (ko) | 2024-01-25 |
| KR20240013283A (ko) | 2024-01-30 |
| US10900034B2 (en) | 2021-01-26 |
| AU2015355546B2 (en) | 2021-10-14 |
| CN107250148B (zh) | 2021-04-16 |
| US10337001B2 (en) | 2019-07-02 |
| US20250092389A1 (en) | 2025-03-20 |
| US20230416733A1 (en) | 2023-12-28 |
| WO2016089433A1 (en) | 2016-06-09 |
| KR102763527B1 (ko) | 2025-02-05 |
| US20210079389A1 (en) | 2021-03-18 |
| JP2022078083A (ja) | 2022-05-24 |
| EP3227447B1 (en) | 2024-04-24 |
| US20180051281A1 (en) | 2018-02-22 |
| CA2969619A1 (en) | 2016-06-09 |
| EP4400584A3 (en) | 2024-10-16 |
| EP3227447A1 (en) | 2017-10-11 |
| JP2017537626A (ja) | 2017-12-21 |
| US20160289675A1 (en) | 2016-10-06 |
| EP3227447A4 (en) | 2018-07-11 |
| KR20170084334A (ko) | 2017-07-19 |
| CN107250148A (zh) | 2017-10-13 |
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