CA2406687A1 - Chimeric promoters for controlling expression in muscle cells - Google Patents
Chimeric promoters for controlling expression in muscle cells Download PDFInfo
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- CA2406687A1 CA2406687A1 CA002406687A CA2406687A CA2406687A1 CA 2406687 A1 CA2406687 A1 CA 2406687A1 CA 002406687 A CA002406687 A CA 002406687A CA 2406687 A CA2406687 A CA 2406687A CA 2406687 A1 CA2406687 A1 CA 2406687A1
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Classifications
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
- 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
- C12N15/86—Viral vectors
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
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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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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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
- C12N2830/80—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
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- Molecular Biology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
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- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
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- Microbiology (AREA)
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- Physics & Mathematics (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP01440378.6 | 2001-11-09 | ||
| EP01440378 | 2001-11-09 | ||
| US33176701P | 2001-11-21 | 2001-11-21 | |
| US60/331,767 | 2001-11-21 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2406687A1 true CA2406687A1 (en) | 2003-05-09 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002406687A Abandoned CA2406687A1 (en) | 2001-11-09 | 2002-11-08 | Chimeric promoters for controlling expression in muscle cells |
Country Status (3)
| Country | Link |
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| US (1) | US20030157064A1 (enExample) |
| JP (1) | JP2003189887A (enExample) |
| CA (1) | CA2406687A1 (enExample) |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3592848A1 (en) | 2017-03-10 | 2020-01-15 | Genethon | Treatment of glycogen storage disease iii |
| EP3749771A1 (en) | 2018-02-07 | 2020-12-16 | Genethon | Hybrid regulatory elements |
| AU2019317754B2 (en) | 2018-08-08 | 2025-07-10 | Association Institut De Myologie | Mini-GDE for the treatment of glycogen storage disease III |
| KR20210153069A (ko) | 2019-04-19 | 2021-12-16 | 제네똥 | 섬유아세포 성장 인자 23 관련 저인산혈증성 질환의 유전자 요법 |
| BR112023000305A2 (pt) * | 2020-07-10 | 2023-03-21 | Genethon | Promotor, sistema de expressão e composição farmacêutica |
| EP4200410A1 (en) | 2020-08-24 | 2023-06-28 | Genethon | C-terminal truncated gde for the treatment of glycogen storage disease iii |
| CN113106094B (zh) * | 2021-04-07 | 2022-10-18 | 四川大学 | 增强型骨骼肌细胞高效特异性启动子、筛选方法及应用 |
| US20240358852A1 (en) | 2021-04-20 | 2024-10-31 | Anjarium Biosciences Ag | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
| JP2025504404A (ja) | 2022-01-14 | 2025-02-12 | アンジャリウム バイオサイエンシズ エージー | Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法 |
| CN114231532B (zh) * | 2022-02-22 | 2022-06-17 | 广州派真生物技术有限公司 | 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用 |
| EP4536816A1 (en) | 2022-06-09 | 2025-04-16 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
| WO2024188913A1 (en) | 2023-03-10 | 2024-09-19 | Genethon | Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter |
| WO2025003477A1 (en) | 2023-06-29 | 2025-01-02 | Genethon | N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUP0001964A3 (en) * | 1997-01-29 | 2002-01-28 | Cornell Res Foundation Inc Ith | Multiple site delivery of adenoviral vector for the induction of angiogenesis |
| ATE366319T1 (de) * | 2000-08-17 | 2007-07-15 | Keiya Ozawa | Adeno-assoziierte virus-vermittelte übertragung von angiogenesefaktoren |
| AU2001296600A1 (en) * | 2000-10-06 | 2002-04-15 | Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
| EP2017338A1 (en) * | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
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2002
- 2002-11-08 US US10/290,327 patent/US20030157064A1/en not_active Abandoned
- 2002-11-08 CA CA002406687A patent/CA2406687A1/en not_active Abandoned
- 2002-11-11 JP JP2002327364A patent/JP2003189887A/ja not_active Withdrawn
Also Published As
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| JP2003189887A (ja) | 2003-07-08 |
| US20030157064A1 (en) | 2003-08-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| FZDE | Discontinued |