JP2019521139A5 - - Google Patents
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- JP2019521139A5 JP2019521139A5 JP2019501575A JP2019501575A JP2019521139A5 JP 2019521139 A5 JP2019521139 A5 JP 2019521139A5 JP 2019501575 A JP2019501575 A JP 2019501575A JP 2019501575 A JP2019501575 A JP 2019501575A JP 2019521139 A5 JP2019521139 A5 JP 2019521139A5
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- 125000000217 alkyl group Chemical group 0.000 claims description 216
- 150000001875 compounds Chemical class 0.000 claims description 109
- 238000010362 genome editing Methods 0.000 claims description 72
- 108090000623 proteins and genes Proteins 0.000 claims description 70
- 210000004027 cell Anatomy 0.000 claims description 64
- 239000013078 crystal Substances 0.000 claims description 58
- 125000001153 fluoro group Chemical group F* 0.000 claims description 54
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims description 54
- 229910052731 fluorine Inorganic materials 0.000 claims description 49
- WNLRTRBMVRJNCN-UHFFFAOYSA-N adipic acid Chemical compound OC(=O)CCCCC(O)=O WNLRTRBMVRJNCN-UHFFFAOYSA-N 0.000 claims description 48
- 102000004169 proteins and genes Human genes 0.000 claims description 42
- 150000007523 nucleic acids Chemical class 0.000 claims description 41
- 150000003839 salts Chemical class 0.000 claims description 41
- 229910052739 hydrogen Inorganic materials 0.000 claims description 40
- 239000001257 hydrogen Substances 0.000 claims description 40
- 239000002773 nucleotide Substances 0.000 claims description 38
- 125000003729 nucleotide group Chemical group 0.000 claims description 38
- 125000000623 heterocyclic group Chemical group 0.000 claims description 36
- 150000002431 hydrogen Chemical class 0.000 claims description 36
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 36
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 32
- 108020004707 nucleic acids Proteins 0.000 claims description 32
- 102000039446 nucleic acids Human genes 0.000 claims description 32
- 108091033409 CRISPR Proteins 0.000 claims description 28
- 239000013598 vector Substances 0.000 claims description 26
- 239000001361 adipic acid Substances 0.000 claims description 25
- 235000011037 adipic acid Nutrition 0.000 claims description 25
- 230000014509 gene expression Effects 0.000 claims description 24
- YZCKVEUIGOORGS-OUBTZVSYSA-N Deuterium Chemical compound [2H] YZCKVEUIGOORGS-OUBTZVSYSA-N 0.000 claims description 22
- 229910052805 deuterium Inorganic materials 0.000 claims description 22
- 230000007018 DNA scission Effects 0.000 claims description 21
- 238000010354 CRISPR gene editing Methods 0.000 claims description 20
- 230000037361 pathway Effects 0.000 claims description 20
- 125000006555 (C3-C5) cycloalkyl group Chemical group 0.000 claims description 18
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 18
- 125000002757 morpholinyl group Chemical group 0.000 claims description 18
- 125000003566 oxetanyl group Chemical group 0.000 claims description 18
- 125000003718 tetrahydrofuranyl group Chemical group 0.000 claims description 18
- 125000001412 tetrahydropyranyl group Chemical group 0.000 claims description 18
- KRKNYBCHXYNGOX-UHFFFAOYSA-N citric acid Chemical compound OC(=O)CC(O)(C(O)=O)CC(O)=O KRKNYBCHXYNGOX-UHFFFAOYSA-N 0.000 claims description 15
- 230000008439 repair process Effects 0.000 claims description 15
- 230000006780 non-homologous end joining Effects 0.000 claims description 14
- 108020004414 DNA Proteins 0.000 claims description 13
- 108020005004 Guide RNA Proteins 0.000 claims description 12
- VZCYOOQTPOCHFL-OWOJBTEDSA-N Fumaric acid Chemical compound OC(=O)\C=C\C(O)=O VZCYOOQTPOCHFL-OWOJBTEDSA-N 0.000 claims description 10
- 239000012190 activator Substances 0.000 claims description 10
- WPYMKLBDIGXBTP-UHFFFAOYSA-N benzoic acid Chemical compound OC(=O)C1=CC=CC=C1 WPYMKLBDIGXBTP-UHFFFAOYSA-N 0.000 claims description 10
- VZCYOOQTPOCHFL-UHFFFAOYSA-N trans-butenedioic acid Natural products OC(=O)C=CC(O)=O VZCYOOQTPOCHFL-UHFFFAOYSA-N 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 10
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 8
- KDYFGRWQOYBRFD-UHFFFAOYSA-N succinic acid Chemical compound OC(=O)CCC(O)=O KDYFGRWQOYBRFD-UHFFFAOYSA-N 0.000 claims description 8
- 230000000295 complement effect Effects 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 239000005711 Benzoic acid Substances 0.000 claims description 5
- OFOBLEOULBTSOW-UHFFFAOYSA-N Propanedioic acid Natural products OC(=O)CC(O)=O OFOBLEOULBTSOW-UHFFFAOYSA-N 0.000 claims description 5
- 235000010233 benzoic acid Nutrition 0.000 claims description 5
- 239000001530 fumaric acid Substances 0.000 claims description 5
- VZCYOOQTPOCHFL-UPHRSURJSA-N maleic acid Chemical compound OC(=O)\C=C/C(O)=O VZCYOOQTPOCHFL-UPHRSURJSA-N 0.000 claims description 5
- 239000011976 maleic acid Substances 0.000 claims description 5
- 101710172824 CRISPR-associated endonuclease Cas9 Proteins 0.000 claims description 4
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 claims description 4
- 102000053602 DNA Human genes 0.000 claims description 4
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 4
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 4
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 4
- 101710163270 Nuclease Proteins 0.000 claims description 4
- 101000910035 Streptococcus pyogenes serotype M1 CRISPR-associated endonuclease Cas9/Csn1 Proteins 0.000 claims description 4
- 230000022131 cell cycle Effects 0.000 claims description 4
- 229940125904 compound 1 Drugs 0.000 claims description 4
- 229940125782 compound 2 Drugs 0.000 claims description 4
- 230000005782 double-strand break Effects 0.000 claims description 4
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 4
- 239000012636 effector Substances 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 238000002703 mutagenesis Methods 0.000 claims description 4
- 231100000350 mutagenesis Toxicity 0.000 claims description 4
- 239000013612 plasmid Substances 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 3
- 241000713666 Lentivirus Species 0.000 claims description 3
- 241000700584 Simplexvirus Species 0.000 claims description 3
- 239000001384 succinic acid Substances 0.000 claims description 3
- 230000002103 transcriptional effect Effects 0.000 claims description 3
- 241000701161 unidentified adenovirus Species 0.000 claims description 3
- 241001430294 unidentified retrovirus Species 0.000 claims description 3
- 108020004635 Complementary DNA Proteins 0.000 claims description 2
- 241000124008 Mammalia Species 0.000 claims description 2
- KDYFGRWQOYBRFD-NUQCWPJISA-N butanedioic acid Chemical compound O[14C](=O)CC[14C](O)=O KDYFGRWQOYBRFD-NUQCWPJISA-N 0.000 claims description 2
- 229940126214 compound 3 Drugs 0.000 claims description 2
- 210000004748 cultured cell Anatomy 0.000 claims description 2
- 230000003247 decreasing effect Effects 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 230000010354 integration Effects 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 108091033319 polynucleotide Proteins 0.000 claims description 2
- 102000040430 polynucleotide Human genes 0.000 claims description 2
- 239000002157 polynucleotide Substances 0.000 claims description 2
- 230000001360 synchronised effect Effects 0.000 claims description 2
- -1 1 Chemical class 0.000 claims 10
- 238000005245 sintering Methods 0.000 claims 4
- GHYOCDFICYLMRF-UTIIJYGPSA-N (2S,3R)-N-[(2S)-3-(cyclopenten-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[[(2S)-2-[(2-morpholin-4-ylacetyl)amino]propanoyl]amino]propanamide Chemical compound C1(=CCCC1)C[C@@H](C(=O)[C@@]1(OC1)C)NC([C@H]([C@@H](C1=CC=C(C=C1)OC)O)NC([C@H](C)NC(CN1CCOCC1)=O)=O)=O GHYOCDFICYLMRF-UTIIJYGPSA-N 0.000 claims 1
- 208000009889 Herpes Simplex Diseases 0.000 claims 1
- 229940125797 compound 12 Drugs 0.000 claims 1
- 230000005764 inhibitory process Effects 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
- 108091006106 transcriptional activators Proteins 0.000 claims 1
- 230000035899 viability Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 54
- 239000003795 chemical substances by application Substances 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 238000002715 modification method Methods 0.000 description 1
- 230000002035 prolonged effect Effects 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000004083 survival effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662361961P | 2016-07-13 | 2016-07-13 | |
| US201662361781P | 2016-07-13 | 2016-07-13 | |
| US62/361,781 | 2016-07-13 | ||
| US62/361,961 | 2016-07-13 | ||
| PCT/US2017/041979 WO2018013840A1 (en) | 2016-07-13 | 2017-07-13 | Methods, compositions and kits for increasing genome editing efficiency |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019521139A JP2019521139A (ja) | 2019-07-25 |
| JP2019521139A5 true JP2019521139A5 (cg-RX-API-DMAC7.html) | 2020-08-20 |
| JP7033583B2 JP7033583B2 (ja) | 2022-03-10 |
Family
ID=59388213
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019501575A Active JP7033583B2 (ja) | 2016-07-13 | 2017-07-13 | ゲノム編集効率を高めるための方法、組成物及びキット |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11124805B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3484870B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP7033583B2 (cg-RX-API-DMAC7.html) |
| CN (1) | CN109863143B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2017295720B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3030783A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2938210T3 (cg-RX-API-DMAC7.html) |
| MA (1) | MA45670A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018013840A1 (cg-RX-API-DMAC7.html) |
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| US10967298B2 (en) | 2012-03-15 | 2021-04-06 | Flodesign Sonics, Inc. | Driver and control for variable impedence load |
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| US9458450B2 (en) | 2012-03-15 | 2016-10-04 | Flodesign Sonics, Inc. | Acoustophoretic separation technology using multi-dimensional standing waves |
| US9950282B2 (en) | 2012-03-15 | 2018-04-24 | Flodesign Sonics, Inc. | Electronic configuration and control for acoustic standing wave generation |
| EP3092049A1 (en) | 2014-01-08 | 2016-11-16 | Flodesign Sonics Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11021699B2 (en) | 2015-04-29 | 2021-06-01 | FioDesign Sonics, Inc. | Separation using angled acoustic waves |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11667911B2 (en) | 2015-09-24 | 2023-06-06 | Editas Medicine, Inc. | Use of exonucleases to improve CRISPR/CAS-mediated genome editing |
| CR20180323A (es) | 2015-11-20 | 2018-08-06 | Idorsia Pharmaceuticals Ltd | Derivados de indol n-sustituídos como moduladores de los receptores de pge2 |
| WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| EP4047092B1 (en) | 2016-04-13 | 2025-07-30 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
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| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| CN109863143B (zh) | 2016-07-13 | 2021-10-15 | 威泰克斯制药公司 | 提高基因组编辑效率的方法、组合物和试剂盒 |
| KR20190127655A (ko) | 2016-10-19 | 2019-11-13 | 프로디자인 소닉스, 인크. | 음향학에 의한 친화성 세포 추출 |
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| KR102626982B1 (ko) | 2017-05-18 | 2024-01-18 | 이도르시아 파마슈티컬스 리미티드 | Pge2 수용체 조절제로서의 피리미딘 유도체 |
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| JP2021507561A (ja) | 2017-12-14 | 2021-02-22 | フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． | 音響トランスデューサドライバ及びコントローラ |
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| EP3740483B1 (en) | 2018-01-17 | 2023-01-11 | Vertex Pharmaceuticals Incorporated | Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency |
| JP2021522819A (ja) * | 2018-05-07 | 2021-09-02 | 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 | インビトロベータ細胞分化に関連する細胞集団および遺伝子発現 |
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| CN110592141B (zh) * | 2018-06-13 | 2023-07-07 | 中国科学院上海有机化学研究所 | 用于调控基因编辑效率的化合物及其应用 |
| CN108949830B (zh) * | 2018-08-03 | 2021-11-26 | 福州大学 | 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法 |
| EP3640329A1 (en) * | 2018-10-18 | 2020-04-22 | Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) | Gene editing based cancer treatment |
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| CN111088290B (zh) * | 2019-12-30 | 2021-10-08 | 同济大学 | 一种杜鹃素在基因编辑中的应用 |
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| CN114717232A (zh) * | 2020-12-22 | 2022-07-08 | 未来智人再生医学研究院(广州)有限公司 | 表达irf-1靶向抑制因子的多能干细胞及其衍生物与应用 |
| CN112750498B (zh) * | 2020-12-30 | 2022-06-24 | 同济大学 | 靶向逆转录引物结合位点从而抑制hiv病毒复制的方法 |
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| TW202246510A (zh) | 2021-02-26 | 2022-12-01 | 美商維泰克斯製藥公司 | 以crispr/slucas9治療第1型肌強直性營養不良之組合物及方法 |
| CN113373148B (zh) * | 2021-06-16 | 2022-08-26 | 中国人民解放军军事科学院军事医学研究院 | 一种调控app表达的靶标位点序列及其在防治ad上的应用 |
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| EP4423278A4 (en) * | 2021-10-29 | 2025-10-22 | Univ Duke | COMPOSITIONS AND SYSTEMS FOR PROGRAMMABLE RNA-BASED CELL EDITING AND METHODS OF MAKING AND USING THE SAME |
| CN114277119B (zh) * | 2021-12-09 | 2023-05-16 | 浙江大学医学院附属邵逸夫医院 | 一种环状RNA circ-Arsb在制备防治骨质疏松产品中的应用 |
| WO2023120530A1 (ja) * | 2021-12-24 | 2023-06-29 | 国立大学法人大阪大学 | 相同組換えを利用したゲノム編集細胞の製造方法 |
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| CN116004542A (zh) * | 2022-09-26 | 2023-04-25 | 苏州吉纳星辰生物技术有限公司 | 一种提高基因敲入效率的方法及其应用 |
| WO2024238691A2 (en) * | 2023-05-15 | 2024-11-21 | The Regents Of The University Of California | Andp gene editing system |
| US20250099613A1 (en) * | 2023-07-26 | 2025-03-27 | University Of Utah Research Foundation | Compositions And Methods For Regulating ZNF865 |
| CN119215053B (zh) * | 2024-12-05 | 2025-04-01 | 南昌大学 | Ye6144在制备治疗乳腺癌引起的骨骼肌萎缩药物中的应用 |
| CN119752995B (zh) * | 2024-12-26 | 2025-11-28 | 中国农业科学院棉花研究所 | GhNFXL1基因在提高棉属植物耐盐性和/或耐冷性中的应用 |
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- 2017-07-13 WO PCT/US2017/041979 patent/WO2018013840A1/en not_active Ceased
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