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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Japanese (ja)
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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)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • 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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Family Applications (1)

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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 (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 (3)

* 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

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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
US20140031417A1 (en) 2011-04-06 2014-01-30 Universite Pierre Et Marie Curie (Paris 6) Skeletal muscle-specific enhancer
MX2014015985A (es) 2012-06-19 2015-07-21 Univ Florida Composiciones y metodos para tratar enfermedades.
EP2885008B1 (en) 2012-08-15 2018-11-07 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
EP3760720A1 (en) 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
IL280443B (en) 2013-03-14 2022-07-01 Sarepta Therapeutics Inc Preparations that skip axon for the treatment of muscular dystrophy
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
AU2016227621C1 (en) 2015-03-05 2020-03-05 Peter Und Traudl Engelhorn-Stiftung Zur Förderung 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
EP4011206B1 (en) 2016-06-21 2023-11-01 The Curators of the University of Missouri Modified dystrophin proteins
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3749756A4 (en) * 2018-02-05 2021-12-08 Audentes Therapeutics, Inc. ELEMENTS OF TRANSCRIPTION REGULATION AND ASSOCIATED USES
CN120665951A (zh) 2018-02-07 2025-09-19 吉尼松公司 杂合调控元件
CN112313331A (zh) 2018-04-27 2021-02-02 沃雅戈治疗公司 用于测量aadc病毒载体效力的方法
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
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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
AU2019381460A1 (en) 2018-11-16 2021-06-17 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting Utrophin gene
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
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KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
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EP4013874A1 (en) 2019-08-16 2022-06-22 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting lama1 gene
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021211753A1 (en) 2020-04-15 2021-10-21 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
WO2022073041A2 (en) 2020-09-30 2022-04-07 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
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Cited By (5)

* 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

Also Published As

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

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