JP2018538003A5 - - Google Patents
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- JP2018538003A5 JP2018538003A5 JP2018533200A JP2018533200A JP2018538003A5 JP 2018538003 A5 JP2018538003 A5 JP 2018538003A5 JP 2018533200 A JP2018533200 A JP 2018533200A JP 2018533200 A JP2018533200 A JP 2018533200A JP 2018538003 A5 JP2018538003 A5 JP 2018538003A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- expression cassette
- vector
- acid expression
- regulatory element
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- 108020004707 nucleic acids Proteins 0.000 claims 19
- 102000039446 nucleic acids Human genes 0.000 claims 19
- 150000007523 nucleic acids Chemical class 0.000 claims 19
- 230000014509 gene expression Effects 0.000 claims 13
- 230000001105 regulatory effect Effects 0.000 claims 9
- 210000002889 endothelial cell Anatomy 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 108700019146 Transgenes Proteins 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 210000004027 cell Anatomy 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 102100036537 von Willebrand factor Human genes 0.000 claims 3
- 102000005666 Apolipoprotein A-I Human genes 0.000 claims 2
- 108010059886 Apolipoprotein A-I Proteins 0.000 claims 2
- 102100022641 Coagulation factor IX Human genes 0.000 claims 2
- 102100023804 Coagulation factor VII Human genes 0.000 claims 2
- 206010012310 Dengue fever Diseases 0.000 claims 2
- 108010076282 Factor IX Proteins 0.000 claims 2
- 108010023321 Factor VII Proteins 0.000 claims 2
- 108010054218 Factor VIII Proteins 0.000 claims 2
- 102000001690 Factor VIII Human genes 0.000 claims 2
- 108010074864 Factor XI Proteins 0.000 claims 2
- 102000008299 Nitric Oxide Synthase Human genes 0.000 claims 2
- 108010021487 Nitric Oxide Synthase Proteins 0.000 claims 2
- 108010000499 Thromboplastin Proteins 0.000 claims 2
- 102000002262 Thromboplastin Human genes 0.000 claims 2
- 102100030951 Tissue factor pathway inhibitor Human genes 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 claims 2
- 108010013555 lipoprotein-associated coagulation inhibitor Proteins 0.000 claims 2
- 230000004614 tumor growth Effects 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 108010047303 von Willebrand Factor Proteins 0.000 claims 2
- 229960001134 von willebrand factor Drugs 0.000 claims 2
- 102100032290 A disintegrin and metalloproteinase with thrombospondin motifs 13 Human genes 0.000 claims 1
- 108091005670 ADAMTS13 Proteins 0.000 claims 1
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 102100029761 Cadherin-5 Human genes 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 102100030563 Coagulation factor XI Human genes 0.000 claims 1
- 208000035473 Communicable disease Diseases 0.000 claims 1
- 208000001490 Dengue Diseases 0.000 claims 1
- 201000011001 Ebola Hemorrhagic Fever Diseases 0.000 claims 1
- 208000030820 Ebola disease Diseases 0.000 claims 1
- 102100037241 Endoglin Human genes 0.000 claims 1
- 102100031759 Endothelial cell-specific chemotaxis regulator Human genes 0.000 claims 1
- 102100033902 Endothelin-1 Human genes 0.000 claims 1
- 101150021185 FGF gene Proteins 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 235000010469 Glycine max Nutrition 0.000 claims 1
- 244000068988 Glycine max Species 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 102100035960 Hedgehog-interacting protein Human genes 0.000 claims 1
- 101710164669 Hedgehog-interacting protein Proteins 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 108090000100 Hepatocyte Growth Factor Proteins 0.000 claims 1
- 102100021866 Hepatocyte growth factor Human genes 0.000 claims 1
- 101000794587 Homo sapiens Cadherin-5 Proteins 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 101000881679 Homo sapiens Endoglin Proteins 0.000 claims 1
- 101000866525 Homo sapiens Endothelial cell-specific chemotaxis regulator Proteins 0.000 claims 1
- 101000925493 Homo sapiens Endothelin-1 Proteins 0.000 claims 1
- 101000962526 Homo sapiens Hyaluronidase-2 Proteins 0.000 claims 1
- 101000599858 Homo sapiens Intercellular adhesion molecule 2 Proteins 0.000 claims 1
- 101000840275 Homo sapiens Interferon alpha-inducible protein 27, mitochondrial Proteins 0.000 claims 1
- 101001116302 Homo sapiens Platelet endothelial cell adhesion molecule Proteins 0.000 claims 1
- 101000753253 Homo sapiens Tyrosine-protein kinase receptor Tie-1 Proteins 0.000 claims 1
- 102100039285 Hyaluronidase-2 Human genes 0.000 claims 1
- 102000004877 Insulin Human genes 0.000 claims 1
- 108090001061 Insulin Proteins 0.000 claims 1
- 206010022489 Insulin Resistance Diseases 0.000 claims 1
- 102100037872 Intercellular adhesion molecule 2 Human genes 0.000 claims 1
- 102100029604 Interferon alpha-inducible protein 27, mitochondrial Human genes 0.000 claims 1
- 102000000422 Matrix Metalloproteinase 3 Human genes 0.000 claims 1
- 108010016160 Matrix Metalloproteinase 3 Proteins 0.000 claims 1
- 102000002274 Matrix Metalloproteinases Human genes 0.000 claims 1
- 108010000684 Matrix Metalloproteinases Proteins 0.000 claims 1
- 206010027476 Metastases Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 101150062285 PGF gene Proteins 0.000 claims 1
- 208000018262 Peripheral vascular disease Diseases 0.000 claims 1
- 102100035194 Placenta growth factor Human genes 0.000 claims 1
- 102100024616 Platelet endothelial cell adhesion molecule Human genes 0.000 claims 1
- 208000009714 Severe Dengue Diseases 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 101710172711 Structural protein Proteins 0.000 claims 1
- 208000007536 Thrombosis Diseases 0.000 claims 1
- 201000007023 Thrombotic Thrombocytopenic Purpura Diseases 0.000 claims 1
- 102100022007 Tyrosine-protein kinase receptor Tie-1 Human genes 0.000 claims 1
- 101150045640 VWF gene Proteins 0.000 claims 1
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims 1
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 claims 1
- 206010047249 Venous thrombosis Diseases 0.000 claims 1
- 108010067390 Viral Proteins Proteins 0.000 claims 1
- 208000027276 Von Willebrand disease Diseases 0.000 claims 1
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 1
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims 1
- 230000003143 atherosclerotic effect Effects 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000009391 cell specific gene expression Effects 0.000 claims 1
- 208000020832 chronic kidney disease Diseases 0.000 claims 1
- 208000022831 chronic renal failure syndrome Diseases 0.000 claims 1
- 229940105774 coagulation factor ix Drugs 0.000 claims 1
- 229940105772 coagulation factor vii Drugs 0.000 claims 1
- 229940105778 coagulation factor viii Drugs 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 208000029078 coronary artery disease Diseases 0.000 claims 1
- 208000025729 dengue disease Diseases 0.000 claims 1
- 201000002950 dengue hemorrhagic fever Diseases 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 229960004222 factor ix Drugs 0.000 claims 1
- 229940012413 factor vii Drugs 0.000 claims 1
- 229960000301 factor viii Drugs 0.000 claims 1
- 239000003102 growth factor Substances 0.000 claims 1
- 208000019622 heart disease Diseases 0.000 claims 1
- 238000009396 hybridization Methods 0.000 claims 1
- 230000006058 immune tolerance Effects 0.000 claims 1
- 230000002163 immunogen Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 229940125396 insulin Drugs 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 208000019423 liver disease Diseases 0.000 claims 1
- 238000013160 medical therapy Methods 0.000 claims 1
- 230000009401 metastasis Effects 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- -1 sFLT1 Proteins 0.000 claims 1
- 230000011664 signaling Effects 0.000 claims 1
- 210000004927 skin cell Anatomy 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 238000011282 treatment Methods 0.000 claims 1
- 230000005747 tumor angiogenesis Effects 0.000 claims 1
- 208000001072 type 2 diabetes mellitus Diseases 0.000 claims 1
- 238000002255 vaccination Methods 0.000 claims 1
- 229960005486 vaccine Drugs 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
- 208000012137 von Willebrand disease (hereditary or acquired) Diseases 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021040766A JP7376018B2 (ja) | 2015-12-22 | 2021-03-12 | 内皮特異的核酸調節エレメントならびにその方法および使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP2015081075 | 2015-12-22 | ||
| EPPCT/EP2015/081075 | 2015-12-22 | ||
| PCT/EP2016/082314 WO2017109039A1 (en) | 2015-12-22 | 2016-12-22 | Endothelium-specific nucleic acid regulatory elements and methods and use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021040766A Division JP7376018B2 (ja) | 2015-12-22 | 2021-03-12 | 内皮特異的核酸調節エレメントならびにその方法および使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018538003A JP2018538003A (ja) | 2018-12-27 |
| JP2018538003A5 true JP2018538003A5 (cg-RX-API-DMAC7.html) | 2020-02-06 |
Family
ID=57821918
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018533200A Pending JP2018538003A (ja) | 2015-12-22 | 2016-12-22 | 内皮特異的核酸調節エレメントならびにその方法および使用 |
| JP2021040766A Active JP7376018B2 (ja) | 2015-12-22 | 2021-03-12 | 内皮特異的核酸調節エレメントならびにその方法および使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021040766A Active JP7376018B2 (ja) | 2015-12-22 | 2021-03-12 | 内皮特異的核酸調節エレメントならびにその方法および使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11446375B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3393523B9 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2018538003A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2016378480A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3009264A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017109039A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IT201700086403A1 (it) * | 2017-07-27 | 2019-01-27 | Univ Del Piemonte Orientale | Endothelial-specific promoter sequences and uses thereof |
| JP2022505876A (ja) | 2018-10-26 | 2022-01-14 | フリーイェ・ユニヴェルシテイト・ブリュッセル | 遺伝子治療を向上させる新しいツール及びその使用 |
| CN114651005A (zh) * | 2019-01-24 | 2022-06-21 | 艾克赛尔吉恩公司 | 生产和使用重组α1-抗胰蛋白酶(AAT)及其组合物的方法 |
| WO2020168362A1 (en) | 2019-02-15 | 2020-08-20 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| CN113710284A (zh) * | 2019-06-05 | 2021-11-26 | 克里斯珀医疗股份公司 | 具有改善的因子viii表达的血友病a基因编辑 |
| CN111534541A (zh) * | 2020-05-07 | 2020-08-14 | 西南大学 | 一种真核生物CRISPR-Cas9双gRNA载体及构建方法 |
| WO2022031760A1 (en) * | 2020-08-05 | 2022-02-10 | Spacecraft Seven, Llc | Adeno-associated viral vector for glut1 expression and uses thereof |
| US20220233714A1 (en) * | 2021-01-27 | 2022-07-28 | Vanderbilt University | Nanogold-dna bioconjugates and methods of use thereof |
| CN114032239B (zh) * | 2021-10-28 | 2024-06-21 | 北京美康基免生物科技有限公司 | 一种组织特异启动子及其应用 |
| EP4368209A1 (en) | 2022-11-09 | 2024-05-15 | Vrije Universiteit Brussel | Cardiac vascular endothelial cell-derived nucleic acid regulatory elements and methods and use thereof |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5656454A (en) * | 1994-10-04 | 1997-08-12 | President And Fellows Of Harvard College | Endothelial cell-specific enhancer |
| FR2756570B1 (fr) * | 1996-12-03 | 2002-09-27 | Commissariat Energie Atomique | Promoteur de la ve-cadherine et ses utilisations |
| AU2003222427B8 (en) * | 2000-11-17 | 2010-04-29 | Vascular Biogenics Ltd. | Promoters exhibiting endothelial cell specificity and methods of using same |
| ITMI20011138A1 (it) * | 2001-05-29 | 2002-11-29 | Uni Di Torino | Vettore e metodi di impiego per l'espressione selettiva di geni in siti di angiogenesi in vivo |
| JP2005204549A (ja) * | 2004-01-21 | 2005-08-04 | Hubit Genomix Inc | Ｃ型肝炎ウイルス感染者の経過に関する遺伝子およびその利用 |
| US20070161031A1 (en) | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| WO2008073303A2 (en) * | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
| US8815779B2 (en) * | 2009-09-16 | 2014-08-26 | SwitchGear Genomics, Inc. | Transcription biomarkers of biological responses and methods |
| US20120301443A1 (en) * | 2009-12-29 | 2012-11-29 | Cornell University | Methods for developing endothelial cells from pluripotent cells and endothelial cells derived |
| WO2012112586A1 (en) * | 2011-02-14 | 2012-08-23 | Revivicor, Inc. | Genetically modified pigs for xenotransplantation of vascularized xenografts and derivatives thereof |
| JP2015109833A (ja) | 2013-11-08 | 2015-06-18 | 学校法人慶應義塾 | 線維芽細胞から血管内皮細胞を製造する方法 |
-
2016
- 2016-12-22 CA CA3009264A patent/CA3009264A1/en active Pending
- 2016-12-22 US US16/065,629 patent/US11446375B2/en active Active
- 2016-12-22 WO PCT/EP2016/082314 patent/WO2017109039A1/en not_active Ceased
- 2016-12-22 EP EP16826729.2A patent/EP3393523B9/en active Active
- 2016-12-22 AU AU2016378480A patent/AU2016378480A1/en not_active Abandoned
- 2016-12-22 JP JP2018533200A patent/JP2018538003A/ja active Pending
-
2021
- 2021-03-12 JP JP2021040766A patent/JP7376018B2/ja active Active
-
2022
- 2022-07-26 US US17/815,136 patent/US20230051499A1/en active Pending
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