DE60231975D1 - Muskelspezifische expressionsvektoren - Google Patents

Muskelspezifische expressionsvektoren

Info

Publication number
DE60231975D1
DE60231975D1 DE60231975T DE60231975T DE60231975D1 DE 60231975 D1 DE60231975 D1 DE 60231975D1 DE 60231975 T DE60231975 T DE 60231975T DE 60231975 T DE60231975 T DE 60231975T DE 60231975 D1 DE60231975 D1 DE 60231975D1
Authority
DE
Germany
Prior art keywords
muscle
promoter
elements
expression vectors
specific expression
Prior art date
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.)
Expired - Lifetime
Application number
DE60231975T
Other languages
English (en)
Inventor
David Souza
Donna Armentano
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of DE60231975D1 publication Critical patent/DE60231975D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • 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)
  • Plant Pathology (AREA)
  • 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)
DE60231975T 2001-05-24 2002-05-22 Muskelspezifische expressionsvektoren Expired - Lifetime DE60231975D1 (de)

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 (1)

Publication Number Publication Date
DE60231975D1 true DE60231975D1 (de) 2009-05-28

Family

ID=23128549

Family Applications (1)

Application Number Title Priority Date Filing Date
DE60231975T Expired - Lifetime DE60231975D1 (de) 2001-05-24 2002-05-22 Muskelspezifische expressionsvektoren

Country Status (7)

Country Link
US (2) US20030100526A1 (de)
EP (2) EP2017338A1 (de)
JP (1) JP2004535801A (de)
AT (1) ATE428777T1 (de)
CA (1) CA2448120A1 (de)
DE (1) DE60231975D1 (de)
WO (1) WO2002095006A2 (de)

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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
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
AU2013277215A1 (en) * 2012-06-19 2015-02-05 University Of Florida Research Foundation, Inc. Compositions and methods for treating diseases
EP2885008B1 (de) 2012-08-15 2018-11-07 University Of Virginia Patent Foundation Zusammensetzungen und verfahren zur behandlung von peripherer arterienerkrankung
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
WO2014153220A2 (en) 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
LT2970964T (lt) 2013-03-14 2019-04-25 Sarepta Therapeutics, Inc. Kompozicijos su egzono praleidimu, skirtos raumenų distrofijos gydymui
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
IL254293B (en) 2015-03-05 2022-09-01 Peter Und Traudl Engelhorn Stiftung Zur Forderung Der Lebenswissenschaften A system for displaying 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
RU2755544C2 (ru) 2016-04-14 2021-09-17 Бенитек Биофарма Лимитед Реагенты для лечения окулофарингеальной мышечной дистрофии (OPMD) и их применение
US11202840B2 (en) 2016-06-21 2021-12-21 The Curators Of The University Of Missouri Modified dystrophin proteins
JP7221275B2 (ja) 2017-08-03 2023-02-13 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を送達するための組成物および方法
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7381494B2 (ja) * 2018-02-05 2023-11-15 オーデンツ セラピューティクス，インコーポレーテッド 転写調節要素及びその使用
JP7467356B2 (ja) * 2018-02-07 2024-04-15 ジェネトン ハイブリッド調節要素
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
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
MA52631A (fr) 2018-05-15 2021-03-24 Voyager Therapeutics Inc Compositions et méthodes pour le traitement de la maladie de parkinson
US20210301305A1 (en) 2018-06-13 2021-09-30 Voyager Therapeutics, Inc. Engineered untranslated regions (utr) for aav production
WO2020010042A1 (en) 2018-07-02 2020-01-09 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
CA3115248A1 (en) 2018-10-05 2020-04-09 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
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
JP7069426B2 (ja) 2018-11-16 2022-05-17 アステラス製薬株式会社 ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CA3130221A1 (en) 2019-02-28 2020-09-03 Benitec IP Holdings Inc. Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)
US20220162640A1 (en) * 2019-04-08 2022-05-26 Genethon Hybrid promoters for muscle expression
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US20220233721A1 (en) 2019-05-28 2022-07-28 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
TW202112797A (zh) 2019-08-16 2021-04-01 日商摩大力斯醫療公司 經由靶向lama1基因治療肌肉萎縮症的方法
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
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
BR112022023106A2 (pt) 2020-05-13 2023-01-17 Voyager Therapeutics Inc Redirecionamento de tropismo de capsídeos de aav
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
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
US20220098615A1 (en) 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
WO2022081776A1 (en) 2020-10-13 2022-04-21 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
AR124143A1 (es) 2020-11-25 2023-02-15 Astellas Pharma Inc Método para tratar la distrofia muscular mediante el direccionamiento al gen dmpk
WO2022164923A1 (en) 2021-01-26 2022-08-04 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
AU2022219031A1 (en) 2021-02-12 2023-09-07 Merand Pharmaceuticals, Inc. Agents, compositions, and methods for the treatment of hypoxia and ischemia-related disorders
EP4301860A1 (de) 2021-03-04 2024-01-10 Kriya Therapeutics, Inc. Virale vektorkonstrukte mit dna zur hemmung toll-ähnlicher rezeptoren und verfahren zur verwendung davon
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
TW202325263A (zh) 2021-09-14 2023-07-01 美商雷納嘉德醫療管理公司 非環狀脂質及其使用方法
WO2023044333A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023133561A1 (en) 2022-01-09 2023-07-13 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-igf-1r antibodies and methods of using the same
WO2023178067A1 (en) * 2022-03-16 2023-09-21 Rutgers, The State University Of New Jersey Controlled muscle‐specific gene delivery
WO2023196931A1 (en) 2022-04-07 2023-10-12 Renagade Therapeutics Management Inc. Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023250388A1 (en) 2022-06-22 2023-12-28 Voyager Therapeutics, Inc. Tau binding compounds
WO2024015329A1 (en) * 2022-07-11 2024-01-18 Vita Therapeutics, Inc. Manufacturing of therapeutic satellite cells for treating muscular dystrophy
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds

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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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US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
EP1071806B1 (de) * 1998-04-24 2004-06-16 University of Florida Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
AU2001261063A1 (en) * 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
US6593110B2 (en) * 2000-05-04 2003-07-15 California Institute Of Technology Checkpoint-activating oligonucleotides

Also Published As

Publication number Publication date
EP1390490B1 (de) 2009-04-15
CA2448120A1 (en) 2002-11-28
JP2004535801A (ja) 2004-12-02
WO2002095006A2 (en) 2002-11-28
US20110212529A1 (en) 2011-09-01
WO2002095006A3 (en) 2003-03-13
EP1390490A2 (de) 2004-02-25
EP1390490A4 (de) 2005-05-11
EP2017338A1 (de) 2009-01-21
US20030100526A1 (en) 2003-05-29
ATE428777T1 (de) 2009-05-15

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