CA2448120A1 - Muscle-specific expression vectors - Google Patents

Muscle-specific expression vectors Download PDF

Info

Publication number
CA2448120A1
CA2448120A1 CA002448120A CA2448120A CA2448120A1 CA 2448120 A1 CA2448120 A1 CA 2448120A1 CA 002448120 A CA002448120 A CA 002448120A CA 2448120 A CA2448120 A CA 2448120A CA 2448120 A1 CA2448120 A1 CA 2448120A1
Authority
CA
Canada
Prior art keywords
promoter
enhancer
muscle
mck
regulatory element
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.)
Abandoned
Application number
CA002448120A
Other languages
English (en)
French (fr)
Inventor
David W. 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
Individual
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 Individual filed Critical Individual
Publication of CA2448120A1 publication Critical patent/CA2448120A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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

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)
  • 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)
  • Physics & Mathematics (AREA)
  • 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)
CA002448120A 2001-05-24 2002-05-22 Muscle-specific expression vectors Abandoned CA2448120A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US29330401P 2001-05-24 2001-05-24
US60/293,304 2001-05-24
PCT/US2002/016748 WO2002095006A2 (en) 2001-05-24 2002-05-22 Muscle-specific expression vectors

Publications (1)

Publication Number Publication Date
CA2448120A1 true CA2448120A1 (en) 2002-11-28

Family

ID=23128549

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002448120A Abandoned CA2448120A1 (en) 2001-05-24 2002-05-22 Muscle-specific expression vectors

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)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
SG11201605906UA (en) 2014-01-21 2016-08-30 Univ Bruxelles Muscle-specific nucleic acid regulatory elements and methods and use thereof
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
HK1257519A1 (zh) * 2015-09-17 2019-10-25 Research Institute At Nationwide Children's Hospital 用於 galgt2 基因治療的方法和物質
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)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019153009A1 (en) 2018-02-05 2019-08-08 Audentes Therapeutics, 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
CA3097192A1 (en) 2018-04-27 2019-10-31 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
EP3793616A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
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
PE20230767A1 (es) 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides 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
CA3192120A1 (en) * 2020-09-11 2022-03-17 James A. Williams Muscle-specific hybrid promoter
US20220098615A1 (en) 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
EP4228610A1 (en) 2020-10-13 2023-08-23 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
WO2022114243A1 (en) 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
EP4284936A1 (en) 2021-01-26 2023-12-06 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
EP4036238A1 (en) * 2021-01-27 2022-08-03 The University Court of the University of Edinburgh Functional nucleic acid molecule and method
WO2022174113A1 (en) 2021-02-12 2022-08-18 Merand Pharmaceuticals, Inc. Agents, compositions, and methods for the treatment of hypoxia and ischemia-related disorders
WO2022187679A1 (en) 2021-03-04 2022-09-09 Kriya Therapeutics, Inc. Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same
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
CA3232386A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
MX2024008509A (es) 2022-01-09 2024-07-19 Kriya Therapeutics Inc Construcciones de vectores para la distribucion de acidos nucleicos que codifican anticuerpos terapeuticos anti-igf-1r y metodos para usarlos.
US20250243510A1 (en) * 2022-03-16 2025-07-31 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
EP4522757A2 (en) 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
US20250243263A1 (en) 2022-06-22 2025-07-31 Voyager Therapeutics, Inc. Tau binding compounds
US20260009003A1 (en) * 2022-07-11 2026-01-08 Vita Therapeutics, Inc. Manufacturing of therapeutic satellite cells for treating muscular dystrophy
AU2023342086A1 (en) 2022-09-15 2025-03-13 Voyager Therapeutics, Inc. Tau binding compounds
WO2025014832A1 (en) 2023-07-07 2025-01-16 Kriya Therapeutics, Inc. Periocular delivery of aav vectors for treating ophthalmic pathologies

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5266488A (en) * 1988-04-06 1993-11-30 The Regents Of The University Of California Troponin T gene promoter and derivatives thereof
US5795872A (en) * 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
ES2219011T3 (es) * 1998-04-24 2004-11-16 University Of Florida Vector viral adeno-asociado recombinante que codifica alfa-1 antitripsina para terapia genica.
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
WO2001083703A2 (en) * 2000-05-04 2001-11-08 California Institute Of Technology Checkpoint-activating oligonucleotides

Also Published As

Publication number Publication date
JP2004535801A (ja) 2004-12-02
EP2017338A1 (en) 2009-01-21
EP1390490A2 (en) 2004-02-25
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

Similar Documents

Publication Publication Date Title
EP1390490B1 (en) Muscle-specific expression vectors
AU2023214366B2 (en) Gene therapies for lysosomal disorders
Tripathy et al. Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus.
CA2684534C (en) Constructs for enhancement of gene expression in muscle
RS20050614A (sr) Ekspresiona kaseta i vektor za prolaznu ili stabilnu ekspresiju egzogenih molekula
EP3924048A1 (en) Myc, cyclin t1 and/or cdk9 for use in the treatment of degenerative heart and cns disorders
KR20220035107A (ko) Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
CN102127546B (zh) 一种骨骼肌特异性actin启动子及其应用
Liu et al. Synthetic promoter for efficient and muscle-specific expression of exogenous genes
KR20140015999A (ko) 신규 MARs 및 이를 이용하여 목적 단백질을 생산하는 방법
AU2002312095A1 (en) Muscle-specific expression vectors
CN114144203A (zh) 源于显性变异基因的疾患的治疗剂
CN106497930B (zh) 骨骼肌细胞高效特异性启动子及其应用
RU2686102C1 (ru) Рекомбинантный вектор для создания плазмидных генетических конструкций, обладающих повышенной длительностью экспрессии целевых генов
Hacobian et al. Pushing the right buttons: Improving efficacy of therapeutic DNA vectors
JP2003503034A (ja) 核内pparレセプターを使用する発現調節システム
CA2834729A1 (en) A skeletal muscle-specific enhancer
US20230141434A1 (en) Direct in vivo reprogramming using transcription factor etv2 gene for endothelial cell and vessel formation
WO2025122739A1 (en) Compositions and methods for treating a heart disease
HK40047428A (en) Gene therapies for lysosomal disorders
EP3087995A1 (en) Mutants of srsf1 (asf/sf2) for treating spinal muscular atrophy
IL135430A (en) Use of a polynucleotide capable of expressing a cytotoxic gene product in the production of a pharmaceutical composition for treating tumors

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued