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 (OSRAM
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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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
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  • Obesity (AREA)
  • Biochemistry (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (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 (OSRAM) 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)

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

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
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
CA2834729A1 (en) 2011-04-06 2012-10-11 Universite Pierre Et Marie Curie (Paris 6) A skeletal muscle-specific enhancer
KR20150020250A (ko) 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
US9845465B2 (en) 2012-08-15 2017-12-19 University Of Virginia Patent Foundation Compositions and methods for treating peripheral arterial disease
WO2014100714A1 (en) 2012-12-20 2014-06-26 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
KR20250047401A (ko) 2013-03-14 2025-04-03 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR20240094032A (ko) 2013-03-14 2024-06-24 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
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
KR102458219B1 (ko) 2015-03-05 2022-10-24 피터 운드 트라우들 앵겔호른-스티프텅 주르 푀르데룽 데르 레벤스위센샤프텐 세포 표면 상에 펩타이드를 제시하기 위한 시스템
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
CA3020754C (en) 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
EP3471544B1 (en) 2016-06-21 2021-12-22 The Curators Of The University Of Missouri Modified dystrophin proteins
CA3071978A1 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
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JP7467356B2 (ja) * 2018-02-07 2024-04-15 ジェネトン ハイブリッド調節要素
CA3097192A1 (en) 2018-04-27 2019-10-31 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
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US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
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US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210395777A1 (en) 2018-10-15 2021-12-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
MX2021005720A (es) 2018-11-16 2021-07-21 Astellas Pharma Inc Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina.
JP2022522995A (ja) 2019-01-18 2022-04-21 ボイジャー セラピューティクス インコーポレイテッド Aav粒子を生成するための方法及びシステム
AU2020229886B2 (en) 2019-02-28 2025-09-18 Benitec IP Holdings Inc. Compositions and methods for treating oculopharyngeal muscular dystrophy (OPMD)
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
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
BR112021023899A2 (pt) 2019-05-28 2022-01-18 Astellas Pharma Inc Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk
KR20220045013A (ko) 2019-08-16 2022-04-12 가부시키가이샤 모달리스 Lama1 유전자를 표적으로 하는 근이영양증의 치료 방법
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
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
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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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CA3226119A1 (en) * 2021-08-04 2023-02-09 Giuseppe RONZITTI Hybrid promoters for gene expression in muscles and in the cns
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
CN118317944A (zh) 2021-09-14 2024-07-09 雷纳嘉德医疗管理公司 非环状脂质及其使用方法
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Cited By (6)

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

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