HUP9904235A2 - Új szerkezetek és vektorok gének célzott és indukálható kifejezésére - Google Patents

Új szerkezetek és vektorok gének célzott és indukálható kifejezésére

Info

Publication number
HUP9904235A2
HUP9904235A2 HU9904235A HUP9904235A HUP9904235A2 HU P9904235 A2 HUP9904235 A2 HU P9904235A2 HU 9904235 A HU9904235 A HU 9904235A HU P9904235 A HUP9904235 A HU P9904235A HU P9904235 A2 HUP9904235 A2 HU P9904235A2
Authority
HU
Hungary
Prior art keywords
genes
vectors
targeted
inducible expression
novel constructs
Prior art date
Application number
HU9904235A
Other languages
English (en)
Inventor
Johan Auwerx
Patrick Benoit
Laurence Berthou
Didier Branellec
Patrice Denefle
Nicolas Duverger
Abderrahim Mahfoudi
Bart Staels
Original Assignee
Rhone-Poulenc Rorer S.A.
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 Rhone-Poulenc Rorer S.A. filed Critical Rhone-Poulenc Rorer S.A.
Publication of HUP9904235A2 publication Critical patent/HUP9904235A2/hu
Publication of HUP9904235A3 publication Critical patent/HUP9904235A3/hu

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
    • 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
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

A találmány tárgyát őlyan új szerkezetek és új vektőrők képezik,melyek gének indűkálható és májspecifikűs kifejezését teszik lehetővé.A találmány tárgyát részletesebben új hibrid prőmótereket, valamintazők alkalmazása képezi gének in vitrő, ex vivő vagy in vivőkifejezésére májsejtekben. A találmány vőnatkőzik még a prőmótert ésegy PPAR aktivátőrt tartalmazó készítményre is. ŕ
HU9904235A 1996-11-08 1997-11-06 Novel constructs and vectors for the targeted and inducible expression of genes HUP9904235A3 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
FR9613691A FR2755699B1 (fr) 1996-11-08 1996-11-08 Nouvelles constructions et vecteurs pour l'expression ciblee et inductible des genes

Publications (2)

Publication Number Publication Date
HUP9904235A2 true HUP9904235A2 (hu) 2000-04-28
HUP9904235A3 HUP9904235A3 (en) 2003-03-28

Family

ID=9497481

Family Applications (1)

Application Number Title Priority Date Filing Date
HU9904235A HUP9904235A3 (en) 1996-11-08 1997-11-06 Novel constructs and vectors for the targeted and inducible expression of genes

Country Status (15)

Country Link
US (1) US20020144302A1 (hu)
EP (1) EP0946740A1 (hu)
JP (1) JP2001503279A (hu)
KR (1) KR20000053163A (hu)
AU (1) AU5057898A (hu)
BR (1) BR9713981A (hu)
CA (1) CA2269864A1 (hu)
CZ (1) CZ163399A3 (hu)
FR (1) FR2755699B1 (hu)
HU (1) HUP9904235A3 (hu)
IL (1) IL129593A0 (hu)
NO (1) NO992153L (hu)
SK (1) SK61499A3 (hu)
WO (1) WO1998021349A1 (hu)
ZA (1) ZA9710114B (hu)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7098025B1 (en) 1997-07-25 2006-08-29 Ligand Pharmaceuticals Incorporated Human peroxisome proliferator activated receptor gamma (pparγ) gene regulatory sequences and uses therefor
FR2795425B1 (fr) * 1999-06-22 2003-12-05 Aventis Pharma Sa Systeme de regulation pharmacologique de l'expression utilisant les recepteurs nucleaires par et leurs ligands
BR0011897A (pt) * 1999-06-22 2002-03-26 Aventis Pharma Sa Composição, vetor, utilização de uma composição, processos para expressão regulada de um ácido nucleico em uma célula, para regulação da expressão de um ácido nucleico e para identificação de ligandos dos ppar, célula, ppar modificado, e, ácido nucleico
FR2806418B1 (fr) * 2000-03-14 2004-07-16 Aventis Pharma Sa Promoteurs hybrides inductibles par l'inflammation, vecteurs les contenant et utilisations
DK1263978T3 (da) * 2000-03-14 2006-04-18 Univ Paris Curie Hybridpromotorer som kan induceres af inflammation, vektorer indeholdende disse og anvendelse deraf
EP1288303A1 (en) * 2001-08-23 2003-03-05 Aventis Pharma S.A. Inducible expression systems employing PPAR transcriptional activators
AU2002333682A1 (en) * 2001-08-02 2003-02-17 Gencell S.A. Inducible expression systems employing ppar transcriptional activators
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2070058A1 (en) * 1991-05-31 1992-12-01 Robert A. Gadski Vectors and methods for assaying the regulation of apolipoprotein ai synthesis
FR2704556B1 (fr) * 1993-04-30 1995-07-13 Rhone Poulenc Rorer Sa Virus recombinants et leur utilisation en thérapie génique.
FR2718329B1 (fr) * 1994-03-21 2002-09-20 Rhone Poulenc Rorer Sa Lapin transgénique sensibilisé aux dyslipoprotéinémies.
FR2726285B1 (fr) * 1994-10-28 1996-11-29 Centre Nat Rech Scient Adenovirus depourvus de particules contaminantes viables, preparation et utilisation

Also Published As

Publication number Publication date
BR9713981A (pt) 2000-05-02
NO992153D0 (no) 1999-05-04
AU5057898A (en) 1998-06-03
SK61499A3 (en) 2000-02-14
IL129593A0 (en) 2000-02-29
JP2001503279A (ja) 2001-03-13
EP0946740A1 (fr) 1999-10-06
HUP9904235A3 (en) 2003-03-28
CZ163399A3 (cs) 1999-08-11
FR2755699B1 (fr) 1998-12-18
WO1998021349A1 (fr) 1998-05-22
ZA9710114B (en) 1998-06-08
FR2755699A1 (fr) 1998-05-15
CA2269864A1 (fr) 1998-05-22
KR20000053163A (ko) 2000-08-25
US20020144302A1 (en) 2002-10-03
NO992153L (no) 1999-05-04

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