CA2448120A1 - Muscle-specific expression vectors - Google Patents
Muscle-specific expression vectors Download PDFInfo
- Publication number
- CA2448120A1 CA2448120A1 CA002448120A CA2448120A CA2448120A1 CA 2448120 A1 CA2448120 A1 CA 2448120A1 CA 002448120 A CA002448120 A CA 002448120A CA 2448120 A CA2448120 A CA 2448120A CA 2448120 A1 CA2448120 A1 CA 2448120A1
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- Prior art keywords
- promoter
- enhancer
- muscle
- mck
- regulatory element
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- 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.)
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- 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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- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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
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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
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- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
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Landscapes
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| US20030157064A1 (en) * | 2001-11-09 | 2003-08-21 | Pascal Neuville | Chimeric promoters for controlling expression in muscle cells |
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| US8557969B2 (en) | 2004-12-01 | 2013-10-15 | The Regents Of The University Of Michigan | Compositions and methods for regulating cardiac performance |
| WO2008054713A2 (en) * | 2006-10-30 | 2008-05-08 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| WO2012136811A1 (en) | 2011-04-06 | 2012-10-11 | Universite Pierre Et Marie Curie (Paris 6) | A skeletal muscle-specific enhancer |
| HK1209633A1 (en) | 2012-06-19 | 2016-04-08 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating diabetes |
| US9845465B2 (en) | 2012-08-15 | 2017-12-19 | University Of Virginia Patent Foundation | Compositions and methods for treating peripheral arterial disease |
| EP3885439A1 (en) | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
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| EP3633035A1 (en) | 2013-03-14 | 2020-04-08 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| US20160220647A1 (en) * | 2013-09-09 | 2016-08-04 | University Of Washington Through Its Center For Commercialization | Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use |
| US11072801B2 (en) * | 2014-01-21 | 2021-07-27 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| JP6876628B2 (ja) | 2015-03-05 | 2021-05-26 | ペーター ウント トラウドル エンゲルホーン−シュティフトゥング ツア フェアデルング デア レーベンスヴィッセンシャフテン | 細胞表面上にペプチドを提示するためのシステム |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN104894651B (zh) * | 2015-06-29 | 2017-04-12 | 天津诺禾医学检验所有限公司 | 微量起始dna的高通量测序文库构建方法及其所构建的高通量测序文库 |
| AU2016323575B2 (en) * | 2015-09-17 | 2022-10-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for GALGT2 gene therapy |
| CA3020754C (en) | 2016-04-14 | 2023-07-25 | Benitec Biopharma Limited | Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof |
| EP4011206B1 (en) | 2016-06-21 | 2023-11-01 | The Curators of the University of Missouri | Modified dystrophin proteins |
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| US5266488A (en) * | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
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| US6461606B1 (en) * | 1998-04-24 | 2002-10-08 | University Of Florida Research Foundation | Materials and methods for gene therapy |
| CN100422320C (zh) * | 2000-04-28 | 2008-10-01 | 肖啸 | 编码抗肌萎缩蛋白小基因的dna序列及其使用方法 |
| WO2001083703A2 (en) * | 2000-05-04 | 2001-11-08 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
-
2002
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 2002-05-22 EP EP02739449A patent/EP1390490B1/en not_active Expired - Lifetime
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-22 AT AT02739449T patent/ATE428777T1/de not_active IP Right Cessation
- 2002-05-22 WO PCT/US2002/016748 patent/WO2002095006A2/en not_active Ceased
- 2002-05-22 DE DE60231975T patent/DE60231975D1/de not_active Expired - Lifetime
- 2002-05-22 EP EP08103273A patent/EP2017338A1/en not_active Withdrawn
- 2002-05-24 US US10/156,604 patent/US20030100526A1/en not_active Abandoned
-
2007
- 2007-03-12 US US11/716,899 patent/US20110212529A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20110212529A1 (en) | 2011-09-01 |
| EP1390490A4 (en) | 2005-05-11 |
| JP2004535801A (ja) | 2004-12-02 |
| ATE428777T1 (de) | 2009-05-15 |
| US20030100526A1 (en) | 2003-05-29 |
| EP2017338A1 (en) | 2009-01-21 |
| EP1390490B1 (en) | 2009-04-15 |
| WO2002095006A3 (en) | 2003-03-13 |
| WO2002095006A2 (en) | 2002-11-28 |
| EP1390490A2 (en) | 2004-02-25 |
| DE60231975D1 (de) | 2009-05-28 |
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| EEER | Examination request | ||
| FZDE | Discontinued |