JP2004535801A - 筋特異的発現ベクター - Google Patents

筋特異的発現ベクター Download PDF

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JP2004535801A
JP2004535801A JP2002592469A JP2002592469A JP2004535801A JP 2004535801 A JP2004535801 A JP 2004535801A JP 2002592469 A JP2002592469 A JP 2002592469A JP 2002592469 A JP2002592469 A JP 2002592469A JP 2004535801 A JP2004535801 A JP 2004535801A
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enhancer
promoter
muscle
regulatory element
mck
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JP2004535801A5 (enExample
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デイビッド ソーザ
ドナ アルメンターノ
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ジェンザイム コーポレイション
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
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  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Obesity (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2002592469A 2001-05-24 2002-05-22 筋特異的発現ベクター Pending JP2004535801A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US29330401P 2001-05-24 2001-05-24
PCT/US2002/016748 WO2002095006A2 (en) 2001-05-24 2002-05-22 Muscle-specific expression vectors

Publications (2)

Publication Number Publication Date
JP2004535801A true JP2004535801A (ja) 2004-12-02
JP2004535801A5 JP2004535801A5 (enExample) 2006-01-05

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JP2002592469A Pending JP2004535801A (ja) 2001-05-24 2002-05-22 筋特異的発現ベクター

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US (2) US20030100526A1 (enExample)
EP (2) EP1390490B1 (enExample)
JP (1) JP2004535801A (enExample)
AT (1) ATE428777T1 (enExample)
CA (1) CA2448120A1 (enExample)
DE (1) DE60231975D1 (enExample)
WO (1) WO2002095006A2 (enExample)

Cited By (4)

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Publication number Priority date Publication date Assignee Title
JP2021168663A (ja) * 2014-01-21 2021-10-28 フレイエ ユニヴェルシテイト ブリュッセルVrije Universiteit Brussel 筋特異的核酸調節エレメント並びにその方法及び使用
JP2022010079A (ja) * 2015-09-17 2022-01-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Galgt2遺伝子治療のための方法および物質
JP2023543154A (ja) * 2020-09-11 2023-10-13 アルデブロン,エル.エル.シー. 筋特異的ハイブリッドプロモーター
JP2024504412A (ja) * 2021-01-27 2024-01-31 ザ・ユニバーシティ・コート・オブ・ザ・ユニバーシティ・オブ・エディンバラ 機能性核酸分子および方法

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US20030157064A1 (en) * 2001-11-09 2003-08-21 Pascal Neuville Chimeric promoters for controlling expression in muscle cells
US20080044393A1 (en) * 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
WO2007046826A2 (en) * 2004-12-01 2007-04-26 The Regents Of The University Of Michigan Compositions and methods for regulating cardiac performance
US8383601B2 (en) * 2006-10-30 2013-02-26 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
CA2834729A1 (en) 2011-04-06 2012-10-11 Universite Pierre Et Marie Curie (Paris 6) A skeletal muscle-specific enhancer
SMT202000445T1 (it) * 2012-06-19 2020-09-10 Univ Florida Composizioni e metodi per trattare patologie
WO2014028762A1 (en) 2012-08-15 2014-02-20 University Of Virginia Patent Foundation Compositions and methods for treating peripheral arterial disease
JP2016502858A (ja) 2012-12-20 2016-02-01 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
EA202090946A3 (ru) 2013-03-14 2021-07-30 Сарепта Терапьютикс, Инк. Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии
HRP20190382T1 (hr) 2013-03-14 2019-04-19 Sarepta Therapeutics, Inc. Pripravci koji preskaču ekson za liječenje mišićne distrofije
WO2015035364A1 (en) * 2013-09-09 2015-03-12 University Of Washington Through Its Center For Commercialization Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use
CA2980819C (en) 2015-03-05 2024-03-05 Peter Und Traudl Engelhorn-Stiftung Zur Forderung Der Lebenswissenschaften System for the presentation of peptides on the cell surface
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
CN109219659B (zh) 2016-04-14 2022-09-09 贝尼泰克生物制药有限公司 用于治疗眼咽型肌营养不良(opmd)的试剂及其用途
US11202840B2 (en) 2016-06-21 2021-12-21 The Curators Of The University Of Missouri Modified dystrophin proteins
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
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BR112022002889A2 (pt) 2019-08-16 2022-05-17 Modalis Therapeutics Corp Método para tratar distrofia muscular tendo como alvo o gene lama1
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
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WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021168663A (ja) * 2014-01-21 2021-10-28 フレイエ ユニヴェルシテイト ブリュッセルVrije Universiteit Brussel 筋特異的核酸調節エレメント並びにその方法及び使用
JP7235806B2 (ja) 2014-01-21 2023-03-08 フレイエ ユニヴェルシテイト ブリュッセル 筋特異的核酸調節エレメント並びにその方法及び使用
JP2023062192A (ja) * 2014-01-21 2023-05-02 フレイエ ユニヴェルシテイト ブリュッセル 筋特異的核酸調節エレメント並びにその方法及び使用
JP2022010079A (ja) * 2015-09-17 2022-01-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Galgt2遺伝子治療のための方法および物質
JP2023543154A (ja) * 2020-09-11 2023-10-13 アルデブロン,エル.エル.シー. 筋特異的ハイブリッドプロモーター
JP7846677B2 (ja) 2020-09-11 2026-04-15 アルデブロン,エル.エル.シー. 筋特異的ハイブリッドプロモーター
JP2024504412A (ja) * 2021-01-27 2024-01-31 ザ・ユニバーシティ・コート・オブ・ザ・ユニバーシティ・オブ・エディンバラ 機能性核酸分子および方法

Also Published As

Publication number Publication date
WO2002095006A2 (en) 2002-11-28
ATE428777T1 (de) 2009-05-15
EP1390490A4 (en) 2005-05-11
DE60231975D1 (de) 2009-05-28
EP1390490B1 (en) 2009-04-15
CA2448120A1 (en) 2002-11-28
WO2002095006A3 (en) 2003-03-13
US20030100526A1 (en) 2003-05-29
EP2017338A1 (en) 2009-01-21
US20110212529A1 (en) 2011-09-01
EP1390490A2 (en) 2004-02-25

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