JP5823969B2 - 心臓特異的核酸調節因子ならびにこの方法および使用 - Google Patents
心臓特異的核酸調節因子ならびにこの方法および使用 Download PDFInfo
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- JP5823969B2 JP5823969B2 JP2012535849A JP2012535849A JP5823969B2 JP 5823969 B2 JP5823969 B2 JP 5823969B2 JP 2012535849 A JP2012535849 A JP 2012535849A JP 2012535849 A JP2012535849 A JP 2012535849A JP 5823969 B2 JP5823969 B2 JP 5823969B2
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cardiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Heart & Thoracic Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP09174519 | 2009-10-29 | ||
| EP09174519.0 | 2009-10-29 | ||
| PCT/EP2010/066474 WO2011051450A1 (en) | 2009-10-29 | 2010-10-29 | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013509168A JP2013509168A (ja) | 2013-03-14 |
| JP2013509168A5 JP2013509168A5 (https=) | 2013-12-19 |
| JP5823969B2 true JP5823969B2 (ja) | 2015-11-25 |
Family
ID=43414855
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012535849A Expired - Fee Related JP5823969B2 (ja) | 2009-10-29 | 2010-10-29 | 心臓特異的核酸調節因子ならびにこの方法および使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US9353164B2 (https=) |
| EP (1) | EP2494058B1 (https=) |
| JP (1) | JP5823969B2 (https=) |
| AU (1) | AU2010311376B2 (https=) |
| CA (1) | CA2778945A1 (https=) |
| WO (1) | WO2011051450A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011051450A1 (en) | 2009-10-29 | 2011-05-05 | Vib Vzw | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
| US8859517B2 (en) | 2011-08-08 | 2014-10-14 | Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione | Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT) |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| WO2014077863A1 (en) * | 2012-11-19 | 2014-05-22 | Nature Technology Corporation | Replicative minicircle vectors with improved expression |
| JP6707028B2 (ja) | 2013-12-09 | 2020-06-10 | ベイラー カレッジ オブ メディスンBaylor College Of Medicine | 心筋細胞新生におけるhippo及びジストロフィン複合体シグナル伝達 |
| PL3097197T3 (pl) | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| US10570183B2 (en) * | 2016-04-19 | 2020-02-25 | The Board Of Regents Of The University Of Texas System | Use of a small native peptide activator of SERCA pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload |
| WO2018178067A1 (en) | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| US20210343368A1 (en) * | 2018-09-05 | 2021-11-04 | Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft | Method for Engineering Synthetic Cis-Regulatory DNA |
| JP7529286B2 (ja) | 2018-10-26 | 2024-08-06 | フリーイェ・ユニヴェルシテイト・ブリュッセル | 肝特異的核酸調節エレメント並びにその方法及び使用 |
| EP4073257A1 (en) * | 2019-12-09 | 2022-10-19 | UCL Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4081643A1 (en) * | 2019-12-24 | 2022-11-02 | Asklepios Biopharmaceutical, Inc. | Regulatory nucleic acid sequences |
| EP4087489B1 (en) | 2020-01-08 | 2025-05-14 | Vitruvia Holdings Inc. | Methods and computing system for processing ultrasound image to determine health of subdermal tissue |
| JP7744027B2 (ja) | 2020-02-18 | 2025-09-25 | フレイエ ユニヴェルシテイト ブリュッセル | 核酸調節エレメントの新規な組合せ並びにその方法及び使用 |
| JP2024502257A (ja) * | 2020-12-21 | 2024-01-18 | フレイエ ユニヴェルシテイト ブリュッセル | 心筋細胞由来核酸調節エレメント並びにその方法及び使用 |
| KR20240032971A (ko) | 2021-07-08 | 2024-03-12 | 테나야 테라퓨틱스, 인코포레이티드 | 유전자 요법을 위한 최적화된 발현 카세트 |
| AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2797273B1 (fr) | 1999-08-06 | 2004-01-02 | Pasteur Institut | Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique |
| JP2003530086A (ja) | 2000-01-14 | 2003-10-14 | ベス イスラエル ディーコネス メディカル センター | 心臓細胞特異的エンハンサー因子とその使用 |
| ATE373095T1 (de) * | 2000-07-14 | 2007-09-15 | Univ Michigan | Mutierte muskelspezifische enhancer |
| US20050004058A1 (en) * | 2000-12-07 | 2005-01-06 | Patrick Benoit | Sequences upstream of the carp gene, vectors containing them and uses thereof |
| US7714100B2 (en) * | 2004-01-27 | 2010-05-11 | Compugen Ltd | Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of cardiac disease |
| BRPI0513897A (pt) * | 2004-07-29 | 2008-05-20 | Stem Cell Innovations Inc | diferenciação de células-tronco |
| US20080081354A1 (en) * | 2006-10-02 | 2008-04-03 | Cardiac Pacemakers, Inc. | Devices, vectors and methods for inducible ischemia cardioprotection |
| WO2008054713A2 (en) | 2006-10-30 | 2008-05-08 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| US20110027234A1 (en) * | 2007-04-11 | 2011-02-03 | Technion Research & Development Foundation Ltd. | Methods for identification and selection of human embryonic stem cell derived cells |
| CA2722238C (en) | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| WO2011051450A1 (en) | 2009-10-29 | 2011-05-05 | Vib Vzw | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
-
2010
- 2010-10-29 WO PCT/EP2010/066474 patent/WO2011051450A1/en not_active Ceased
- 2010-10-29 US US13/504,614 patent/US9353164B2/en not_active Expired - Fee Related
- 2010-10-29 AU AU2010311376A patent/AU2010311376B2/en not_active Ceased
- 2010-10-29 JP JP2012535849A patent/JP5823969B2/ja not_active Expired - Fee Related
- 2010-10-29 CA CA2778945A patent/CA2778945A1/en not_active Abandoned
- 2010-10-29 EP EP10771459.4A patent/EP2494058B1/en not_active Not-in-force
Also Published As
| Publication number | Publication date |
|---|---|
| EP2494058B1 (en) | 2017-04-05 |
| JP2013509168A (ja) | 2013-03-14 |
| EP2494058A1 (en) | 2012-09-05 |
| CA2778945A1 (en) | 2011-05-05 |
| US9353164B2 (en) | 2016-05-31 |
| AU2010311376A1 (en) | 2012-05-24 |
| AU2010311376A2 (en) | 2012-07-12 |
| WO2011051450A1 (en) | 2011-05-05 |
| US20120252882A1 (en) | 2012-10-04 |
| AU2010311376B2 (en) | 2014-07-03 |
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