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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Publication number
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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Japanese (ja)
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JP2004535801A5 (cg-RX-API-DMAC7.html
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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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Publication of JP2004535801A5 publication Critical patent/JP2004535801A5/ja
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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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Obesity (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
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  • Microbiology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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 (cg-RX-API-DMAC7.html) 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

Country Status (7)

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US (2) US20030100526A1 (cg-RX-API-DMAC7.html)
EP (2) EP2017338A1 (cg-RX-API-DMAC7.html)
JP (1) JP2004535801A (cg-RX-API-DMAC7.html)
AT (1) ATE428777T1 (cg-RX-API-DMAC7.html)
CA (1) CA2448120A1 (cg-RX-API-DMAC7.html)
DE (1) DE60231975D1 (cg-RX-API-DMAC7.html)
WO (1) WO2002095006A2 (cg-RX-API-DMAC7.html)

Cited By (4)

* 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
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
US8557969B2 (en) 2004-12-01 2013-10-15 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
EP3818991A3 (en) 2012-06-19 2021-07-14 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
US9845465B2 (en) 2012-08-15 2017-12-19 University Of Virginia Patent Foundation Compositions and methods for treating peripheral arterial disease
AU2013364158A1 (en) 2012-12-20 2015-07-09 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
HRP20190382T1 (hr) 2013-03-14 2019-04-19 Sarepta Therapeutics, Inc. Pripravci koji preskaču ekson za liječenje mišićne distrofije
BR112015023001B8 (pt) 2013-03-14 2022-08-09 Sarepta Therapeutics Inc Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
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
WO2016139354A1 (de) 2015-03-05 2016-09-09 Peter Und Traudl Engelhorn-Stiftung Zur Förderung Der Lebenswissenschaften System zur präsentation von peptiden auf der zelloberfläche
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
SG11201808981XA (en) 2016-04-14 2018-11-29 Benitec Biopharma Ltd Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
US11202840B2 (en) 2016-06-21 2021-12-21 The Curators Of The University Of Missouri Modified dystrophin proteins
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US12296026B2 (en) 2018-02-05 2025-05-13 Astellas Gene Therapies, Inc. Transcription regulatory elements and uses thereof
JP7467356B2 (ja) 2018-02-07 2024-04-15 ジェネトン ハイブリッド調節要素
US12129476B2 (en) 2018-04-27 2024-10-29 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
CN112567035A (zh) 2018-07-02 2021-03-26 沃雅戈治疗公司 肌萎缩侧索硬化症及脊髓相关病症的治疗
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
US20210348194A1 (en) 2018-10-05 2021-11-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター
BR112021009481A2 (pt) 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
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EP3952920A1 (en) * 2019-04-08 2022-02-16 Genethon Hybrid promoters for muscle expression
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3976785A1 (en) 2019-05-28 2022-04-06 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
AU2020334462A1 (en) 2019-08-16 2022-02-24 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting LAMA1 gene
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4126910A1 (en) 2020-04-01 2023-02-08 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
EP4135841A1 (en) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau binding compounds
KR20230022175A (ko) 2020-05-13 2023-02-14 보이저 테라퓨틱스, 인크. Aav 캡시드의 향성 방향변경
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20220098615A1 (en) 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
JP2023548746A (ja) 2020-10-13 2023-11-21 クリヤ セラピューティクス インコーポレイテッド サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用
AR124143A1 (es) 2020-11-25 2023-02-15 Astellas Pharma Inc Método para tratar la distrofia muscular mediante el direccionamiento al gen dmpk
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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
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
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
DE60231975D1 (de) 2009-05-28
US20110212529A1 (en) 2011-09-01
EP1390490A4 (en) 2005-05-11
CA2448120A1 (en) 2002-11-28
EP1390490B1 (en) 2009-04-15
EP1390490A2 (en) 2004-02-25
US20030100526A1 (en) 2003-05-29
EP2017338A1 (en) 2009-01-21
WO2002095006A2 (en) 2002-11-28
ATE428777T1 (de) 2009-05-15
WO2002095006A3 (en) 2003-03-13

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