JP2018527013A5 - - Google Patents
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- JP2018527013A5 JP2018527013A5 JP2018515082A JP2018515082A JP2018527013A5 JP 2018527013 A5 JP2018527013 A5 JP 2018527013A5 JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018527013 A5 JP2018527013 A5 JP 2018527013A5
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- Prior art keywords
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- Prior art date
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- 150000001875 compounds Chemical class 0.000 claims 27
- 239000000178 monomer Substances 0.000 claims 11
- 239000002773 nucleotide Substances 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 230000005782 double-strand break Effects 0.000 claims 6
- 108091033409 CRISPR Proteins 0.000 claims 5
- 238000010354 CRISPR gene editing Methods 0.000 claims 5
- 108020004414 DNA Proteins 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 108700028369 Alleles Proteins 0.000 claims 4
- 108091028113 Trans-activating crRNA Proteins 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 2
- JCAQMQLAHNGVPY-UUOKFMHZSA-N [(2r,3s,4r,5r)-3,4-dihydroxy-5-(2,2,4-trioxo-1h-imidazo[4,5-c][1,2,6]thiadiazin-7-yl)oxolan-2-yl]methyl dihydrogen phosphate Chemical class O[C@@H]1[C@H](O)[C@@H](COP(O)(O)=O)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 JCAQMQLAHNGVPY-UUOKFMHZSA-N 0.000 claims 2
- 238000010362 genome editing Methods 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- GZEFTKHSACGIBG-UGKPPGOTSA-N 1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)-2-propyloxolan-2-yl]pyrimidine-2,4-dione Chemical compound C1=CC(=O)NC(=O)N1[C@]1(CCC)O[C@H](CO)[C@@H](O)[C@H]1O GZEFTKHSACGIBG-UGKPPGOTSA-N 0.000 claims 1
- AGFIRQJZCNVMCW-UAKXSSHOSA-N 5-bromouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(Br)=C1 AGFIRQJZCNVMCW-UAKXSSHOSA-N 0.000 claims 1
- ASUCSHXLTWZYBA-UMMCILCDSA-N 8-Bromoguanosine Chemical compound C1=2NC(N)=NC(=O)C=2N=C(Br)N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O ASUCSHXLTWZYBA-UMMCILCDSA-N 0.000 claims 1
- HDZZVAMISRMYHH-UHFFFAOYSA-N 9beta-Ribofuranosyl-7-deazaadenin Natural products C1=CC=2C(N)=NC=NC=2N1C1OC(CO)C(O)C1O HDZZVAMISRMYHH-UHFFFAOYSA-N 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- 102100021663 Baculoviral IAP repeat-containing protein 5 Human genes 0.000 claims 1
- 102100033245 Cyclin-dependent kinase 16 Human genes 0.000 claims 1
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 1
- 125000000824 D-ribofuranosyl group Chemical group [H]OC([H])([H])[C@@]1([H])OC([H])(*)[C@]([H])(O[H])[C@]1([H])O[H] 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000944357 Homo sapiens Cyclin-dependent kinase 16 Proteins 0.000 claims 1
- 108020005196 Mitochondrial DNA Proteins 0.000 claims 1
- VQAYFKKCNSOZKM-IOSLPCCCSA-N N(6)-methyladenosine Chemical class C1=NC=2C(NC)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O VQAYFKKCNSOZKM-IOSLPCCCSA-N 0.000 claims 1
- 241000588650 Neisseria meningitidis Species 0.000 claims 1
- 108010017324 STAT3 Transcription Factor Proteins 0.000 claims 1
- 102100024040 Signal transducer and activator of transcription 3 Human genes 0.000 claims 1
- 241000193998 Streptococcus pneumoniae Species 0.000 claims 1
- 241000193996 Streptococcus pyogenes Species 0.000 claims 1
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 1
- 102100038836 Superoxide dismutase [Cu-Zn] Human genes 0.000 claims 1
- 108010002687 Survivin Proteins 0.000 claims 1
- 125000003342 alkenyl group Chemical group 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000000304 alkynyl group Chemical group 0.000 claims 1
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 1
- 239000005547 deoxyribonucleotide Substances 0.000 claims 1
- 229910052736 halogen Inorganic materials 0.000 claims 1
- 102000008371 intracellularly ATP-gated chloride channel activity proteins Human genes 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 102200150628 rs151220873 Human genes 0.000 claims 1
- 102200078754 rs863223435 Human genes 0.000 claims 1
- 229940031000 streptococcus pneumoniae Drugs 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- HDZZVAMISRMYHH-KCGFPETGSA-N tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O HDZZVAMISRMYHH-KCGFPETGSA-N 0.000 claims 1
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 1
- 229940045145 uridine Drugs 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562221407P | 2015-09-21 | 2015-09-21 | |
| US62/221,407 | 2015-09-21 | ||
| US201662330827P | 2016-05-02 | 2016-05-02 | |
| US62/330,827 | 2016-05-02 | ||
| PCT/US2016/052889 WO2017053431A2 (en) | 2015-09-21 | 2016-09-21 | Allele selective gene editing and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018527013A JP2018527013A (ja) | 2018-09-20 |
| JP2018527013A5 true JP2018527013A5 (enExample) | 2019-10-31 |
| JP6799058B2 JP6799058B2 (ja) | 2020-12-09 |
Family
ID=58276126
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018515082A Active JP6799058B2 (ja) | 2015-09-21 | 2016-09-21 | アレル選択的な遺伝子編集およびその使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10369232B2 (enExample) |
| EP (1) | EP3353298B1 (enExample) |
| JP (1) | JP6799058B2 (enExample) |
| CN (1) | CN108350454B (enExample) |
| CA (1) | CA3002647A1 (enExample) |
| ES (1) | ES2959815T3 (enExample) |
| WO (1) | WO2017053431A2 (enExample) |
Families Citing this family (59)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | 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 |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| KR102209636B1 (ko) | 2014-02-11 | 2021-01-29 | 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 | Crispr 이용의 다중화된 게놈 조작 |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| DK3234133T3 (da) | 2014-12-18 | 2021-02-08 | Integrated Dna Tech Inc | Crispr-baserede sammensætninger og fremgangsmåder til anvendelse |
| CA2990699A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| 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 |
| AU2017281497B2 (en) | 2016-06-22 | 2023-04-06 | Proqr Therapeutics Ii B.V. | Single-stranded RNA-editing oligonucleotides |
| DK3474669T3 (da) | 2016-06-24 | 2022-06-27 | Univ Colorado Regents | Fremgangsmåde til generering af stregkodede kombinatoriske biblioteker |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| JP7244922B2 (ja) | 2016-09-01 | 2023-03-23 | 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 | 化学修飾された一本鎖ｒｎａ編集オリゴヌクレオチド |
| CN118726313A (zh) | 2016-10-07 | 2024-10-01 | 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 |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| WO2018098383A1 (en) | 2016-11-22 | 2018-05-31 | Integrated Dna Technologies, Inc. | Crispr/cpf1 systems and methods |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| CN108977442B (zh) * | 2017-06-05 | 2023-01-06 | 广州市锐博生物科技有限公司 | 用于dna编辑的系统及其应用 |
| US9982279B1 (en) | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| US10011849B1 (en) | 2017-06-23 | 2018-07-03 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| SG11202002130WA (en) * | 2017-09-28 | 2020-04-29 | Toolgen Inc | Artificial genome manipulation for gene expression regulation |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| AU2018338787C1 (en) * | 2017-09-29 | 2025-10-23 | Intellia Therapeutics, Inc. | Compositions and methods for TTR gene editing and treating ATTR amyloidosis |
| CA3071712C (en) | 2017-09-29 | 2023-02-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus and methods of use |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12227776B2 (en) | 2018-06-13 | 2025-02-18 | Caribou Biosciences, Inc. | Engineered cascade components and cascade complexes |
| US10227576B1 (en) | 2018-06-13 | 2019-03-12 | Caribou Biosciences, Inc. | Engineered cascade components and cascade complexes |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| EP3908257A4 (en) * | 2019-01-08 | 2022-11-09 | Asklepios Biopharmaceutical, Inc. | METHOD FOR THE TREATMENT OF CYSTIC FIBROSIS |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| GB201901873D0 (en) * | 2019-02-11 | 2019-04-03 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for nucleic acid editing |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| JP7610339B2 (ja) | 2019-06-04 | 2025-01-08 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ベータスリップ変異を有するヒト化ｔｔｒ遺伝子座を含む非ヒト動物と使用方法 |
| WO2021050593A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of sod1 |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| GB2614813B (en) | 2020-05-08 | 2025-05-07 | Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2025166325A1 (en) * | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | MODIFIED GUIDE RNAs |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2162538T3 (pl) * | 2007-05-22 | 2016-10-31 | Oligomery do zastosowań terapeutycznych | |
| PL3494997T3 (pl) * | 2012-07-25 | 2020-04-30 | The Broad Institute, Inc. | Indukowalne białka wiążące dna i narzędzia perturbacji genomu oraz ich zastosowania |
| IL307735A (en) * | 2012-12-12 | 2023-12-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| US9957515B2 (en) * | 2013-03-15 | 2018-05-01 | Cibus Us Llc | Methods and compositions for targeted gene modification |
| WO2014186585A2 (en) * | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| CA2915845A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| AU2014281026B2 (en) * | 2013-06-17 | 2020-05-28 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015048577A2 (en) | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| AU2014348212C1 (en) | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| EP3470089A1 (en) * | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
-
2016
- 2016-09-21 ES ES16849508T patent/ES2959815T3/es active Active
- 2016-09-21 EP EP16849508.3A patent/EP3353298B1/en active Active
- 2016-09-21 CN CN201680067107.5A patent/CN108350454B/zh active Active
- 2016-09-21 WO PCT/US2016/052889 patent/WO2017053431A2/en not_active Ceased
- 2016-09-21 CA CA3002647A patent/CA3002647A1/en active Pending
- 2016-09-21 US US15/272,107 patent/US10369232B2/en active Active
- 2016-09-21 JP JP2018515082A patent/JP6799058B2/ja active Active
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