JP2003189887A - 筋肉細胞での発現を制御するためのキメラプロモーター - Google Patents

筋肉細胞での発現を制御するためのキメラプロモーター

Info

Publication number
JP2003189887A
JP2003189887A JP2002327364A JP2002327364A JP2003189887A JP 2003189887 A JP2003189887 A JP 2003189887A JP 2002327364 A JP2002327364 A JP 2002327364A JP 2002327364 A JP2002327364 A JP 2002327364A JP 2003189887 A JP2003189887 A JP 2003189887A
Authority
JP
Japan
Prior art keywords
gene
human
vector
expression
skeletal
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.)
Withdrawn
Application number
JP2002327364A
Other languages
English (en)
Japanese (ja)
Other versions
JP2003189887A5 (enExample
Inventor
Pascal Neuville
パスカル、ヌビル
Sebastien Ribault
セバッチャン、リボー
Valerie Calenda
バレリ、カランダ
Melanie Frauli
メラニ、フローリ
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 JP2003189887A publication Critical patent/JP2003189887A/ja
Publication of JP2003189887A5 publication Critical patent/JP2003189887A5/ja
Withdrawn legal-status Critical Current

Links

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)
JP2002327364A 2001-11-09 2002-11-11 筋肉細胞での発現を制御するためのキメラプロモーター Withdrawn JP2003189887A (ja)

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
US60/331767 2001-11-21

Publications (2)

Publication Number Publication Date
JP2003189887A true JP2003189887A (ja) 2003-07-08
JP2003189887A5 JP2003189887A5 (enExample) 2005-10-13

Family

ID=26077288

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2002327364A Withdrawn JP2003189887A (ja) 2001-11-09 2002-11-11 筋肉細胞での発現を制御するためのキメラプロモーター

Country Status (3)

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

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113106094A (zh) * 2021-04-07 2021-07-13 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
JP2023532806A (ja) * 2020-07-10 2023-07-31 ジェネトン 新規の筋肉特異的プロモーター
JP7906715B2 (ja) 2020-07-10 2026-08-18 ジェネトン 新規の筋肉特異的プロモーター

Families Citing this family (11)

* 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
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

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023532806A (ja) * 2020-07-10 2023-07-31 ジェネトン 新規の筋肉特異的プロモーター
JP7906715B2 (ja) 2020-07-10 2026-08-18 ジェネトン 新規の筋肉特異的プロモーター
CN113106094A (zh) * 2021-04-07 2021-07-13 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
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

Also Published As

Publication number Publication date
CA2406687A1 (en) 2003-05-09
US20030157064A1 (en) 2003-08-21

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