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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- 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 (cg-RX-API-DMAC7.html) | 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 (6)
| Country | Link |
|---|---|
| US (1) | US10369232B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3353298B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP6799058B2 (cg-RX-API-DMAC7.html) |
| CN (1) | CN108350454B (cg-RX-API-DMAC7.html) |
| ES (1) | ES2959815T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017053431A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (59)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | 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 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| WO2015123339A1 (en) | 2014-02-11 | 2015-08-20 | The Regents Of The University Of Colorado, A Body Corporate | Crispr enabled multiplexed genome engineering |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| EP3786296A1 (en) | 2014-12-18 | 2021-03-03 | Integrated Dna Technologies, Inc. | Crispr-based compositions and methods of use |
| 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 |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| US10017760B2 (en) | 2016-06-24 | 2018-07-10 | Inscripta, Inc. | Methods for generating barcoded combinatorial libraries |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| 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 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| 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 |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| KR20250021632A (ko) | 2016-11-22 | 2025-02-13 | 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. | Crispr/cpf1 시스템 및 방법 |
| 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | 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 |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | 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 |
| 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 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| CN118581083A (zh) * | 2017-09-28 | 2024-09-03 | 株式会社图尔金 | 用于基因表达调节的人工基因组操纵 |
| EP4276185A3 (en) | 2017-09-29 | 2024-02-21 | Regeneron Pharmaceuticals, Inc. | Rodents comprising a humanized ttr locus and methods of use |
| BR112020005287A2 (pt) * | 2017-09-29 | 2020-09-24 | Intellia Therapeutics, Inc. | composições e métodos para edição de gene ttr e tratar amiloidose attr |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US10227576B1 (en) | 2018-06-13 | 2019-03-12 | Caribou Biosciences, Inc. | Engineered cascade components and cascade complexes |
| US12227776B2 (en) | 2018-06-13 | 2025-02-18 | Caribou Biosciences, Inc. | Engineered cascade components and cascade complexes |
| CA3104856A1 (en) | 2018-06-29 | 2020-01-02 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| CA3125924A1 (en) * | 2019-01-08 | 2020-07-16 | Asklepios Biopharmaceutical, Inc. | A method of treating 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 |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US11891618B2 (en) | 2019-06-04 | 2024-02-06 | Regeneron Pharmaceuticals, Inc. | Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| US20220348925A1 (en) | 2019-09-09 | 2022-11-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of sod1 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| 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 |
|---|---|---|---|---|
| KR101629017B1 (ko) * | 2007-05-22 | 2016-06-10 | 아크투루스 쎄라퓨틱스, 인크. | 히드록시메틸 치환된 ｒｎａ 올리고뉴클레오티드 및 ｒｎａ 복합체 |
| EP3808844A1 (en) * | 2012-07-25 | 2021-04-21 | The Broad Institute, Inc. | Inducible dna binding proteins and genome perturbation tools and applications thereof |
| PL2771468T3 (pl) * | 2012-12-12 | 2015-07-31 | Broad Inst Inc | Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami |
| 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 |
| CN106062197A (zh) * | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
| EP3620524A1 (en) * | 2013-06-17 | 2020-03-11 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| 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 |
| WO2015089419A2 (en) * | 2013-12-12 | 2015-06-18 | 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 EP EP16849508.3A patent/EP3353298B1/en active Active
- 2016-09-21 JP JP2018515082A patent/JP6799058B2/ja active Active
- 2016-09-21 ES ES16849508T patent/ES2959815T3/es 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 US US15/272,107 patent/US10369232B2/en active Active
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