CA2406687A1 - Chimeric promoters for controlling expression in muscle cells - Google Patents

Chimeric promoters for controlling expression in muscle cells Download PDF

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Publication number
CA2406687A1
CA2406687A1 CA002406687A CA2406687A CA2406687A1 CA 2406687 A1 CA2406687 A1 CA 2406687A1 CA 002406687 A CA002406687 A CA 002406687A CA 2406687 A CA2406687 A CA 2406687A CA 2406687 A1 CA2406687 A1 CA 2406687A1
Authority
CA
Canada
Prior art keywords
gene
expression
vector
human
cells
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
CA002406687A
Other languages
English (en)
French (fr)
Inventor
Pascal Neuville
Sebastien Ribault
Valerie Calenda
Melanie Frauli
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.)
Transgene SA
Original Assignee
Transgene SA
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 Transgene SA filed Critical Transgene SA
Publication of CA2406687A1 publication Critical patent/CA2406687A1/en
Abandoned legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular 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)
CA002406687A 2001-11-09 2002-11-08 Chimeric promoters for controlling expression in muscle cells Abandoned CA2406687A1 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP01440378.6 2001-11-09
EP01440378 2001-11-09
US33176701P 2001-11-21 2001-11-21
US60/331,767 2001-11-21

Publications (1)

Publication Number Publication Date
CA2406687A1 true CA2406687A1 (en) 2003-05-09

Family

ID=26077288

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002406687A Abandoned CA2406687A1 (en) 2001-11-09 2002-11-08 Chimeric promoters for controlling expression in muscle cells

Country Status (3)

Country Link
US (1) US20030157064A1 (enExample)
JP (1) JP2003189887A (enExample)
CA (1) CA2406687A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA47820A (fr) 2017-03-10 2020-01-15 Genethon Traitement de la glycogénose de type iii
CA3086942A1 (en) 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
CA3107572A1 (en) 2018-08-08 2020-02-13 Genethon Mini-gde for the treatment of glycogen storage disease iii
EP3956354A1 (en) 2019-04-19 2022-02-23 Genethon Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases
JP2023539219A (ja) 2020-08-24 2023-09-13 ジェネトン 糖原病iiiの治療のためのc末端切断gde
CN113106094B (zh) * 2021-04-07 2022-10-18 四川大学 增强型骨骼肌细胞高效特异性启动子、筛选方法及应用
JP2024517427A (ja) 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ-α-1,6-グルコシダーゼ、4-α-グルカノトランスフェラーゼをコードするDNA分子の組成物、その作製の方法、及びその使用の方法
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
CN114231532B (zh) * 2022-02-22 2022-06-17 广州派真生物技术有限公司 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用
WO2023237731A1 (en) 2022-06-09 2023-12-14 Genethon N-terminal truncated gde for the treatment of glycogen storage disease iii
WO2024188913A1 (en) 2023-03-10 2024-09-19 Genethon Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter
WO2025003477A1 (en) 2023-06-29 2025-01-02 Genethon N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR9806819A (pt) * 1997-01-29 2000-05-09 Cornell Res Foundation Inc Fornecimento de múltiplos locais de vetor adenoviral para a indução de angiogênese.
ES2288993T3 (es) * 2000-08-17 2008-02-01 Keiya Ozawa Distribucion de factores angiogenicos mediada por virus adenoasociados.
EP1366160B1 (en) * 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors

Also Published As

Publication number Publication date
US20030157064A1 (en) 2003-08-21
JP2003189887A (ja) 2003-07-08

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Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued