BR9713981A - Vetor recombinante para a expressão induzìvel e hepatoespecìfica de uma molécula, adenovìrus recombinante, vetor adenoviral, variante do promotor do gene da apolipoproteìna aii humana, célula, composição farmacêutica, processo para produção de uma proteìna recombinante, e, utilização de um adenovìrus - Google Patents

Vetor recombinante para a expressão induzìvel e hepatoespecìfica de uma molécula, adenovìrus recombinante, vetor adenoviral, variante do promotor do gene da apolipoproteìna aii humana, célula, composição farmacêutica, processo para produção de uma proteìna recombinante, e, utilização de um adenovìrus

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Publication number
BR9713981A
BR9713981A BR9713981-5A BR9713981A BR9713981A BR 9713981 A BR9713981 A BR 9713981A BR 9713981 A BR9713981 A BR 9713981A BR 9713981 A BR9713981 A BR 9713981A
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BR
Brazil
Prior art keywords
recombinant
adenovirus
vector
variant
molecule
Prior art date
Application number
BR9713981-5A
Other languages
English (en)
Inventor
Abderrahim Mahfoudi
Patrick Benoit
Didier Branellec
Patrice Denefle
Nicolas Duverger
Laurence Berthou
Johan Auwerx
Bart Staels
Original Assignee
Rhone Poulenc Rorer 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 Rhone Poulenc Rorer Sa filed Critical Rhone Poulenc Rorer Sa
Publication of BR9713981A publication Critical patent/BR9713981A/pt

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    • 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
    • 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
    • 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

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  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (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)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

"VETOR RECOMBINANTE PARA A EXPRESSãO INDUZìVEL E HEPATOESPECìFICA DE UMA MOLéCULA, ADENOVìRUS RECOMBINANTE, VETOR ADENOVIRAL, VARIANTE DO PROMOTOR DO GENE DA APOLIPOPROTEìNA AII HUMANA, CéLULA, COMPOSIçãO FARMACêUTICA, PROCESSO PARA PRODUçãO DE UMA PROTEìNA RECOMBINANTE, E, UTILIZAçãO DE UM ADENOVìRUS". A presente invenção refere-se a novas construções e a novos vetores que permitem uma expressão direcionada e indutível de genes. Ela descreve em particular novos promotores híbridos e sua utlização para a expressão de genes nas células hepáticas, in vitro, ex vivo ou in vivo.
BR9713981-5A 1996-11-08 1997-11-06 Vetor recombinante para a expressão induzìvel e hepatoespecìfica de uma molécula, adenovìrus recombinante, vetor adenoviral, variante do promotor do gene da apolipoproteìna aii humana, célula, composição farmacêutica, processo para produção de uma proteìna recombinante, e, utilização de um adenovìrus BR9713981A (pt)

Applications Claiming Priority (2)

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
PCT/FR1997/001992 WO1998021349A1 (fr) 1996-11-08 1997-11-06 Nouvelles constructions et vecteurs pour l'expression ciblee et inductible de genes

Publications (1)

Publication Number Publication Date
BR9713981A true BR9713981A (pt) 2000-05-02

Family

ID=9497481

Family Applications (1)

Application Number Title Priority Date Filing Date
BR9713981-5A BR9713981A (pt) 1996-11-08 1997-11-06 Vetor recombinante para a expressão induzìvel e hepatoespecìfica de uma molécula, adenovìrus recombinante, vetor adenoviral, variante do promotor do gene da apolipoproteìna aii humana, célula, composição farmacêutica, processo para produção de uma proteìna recombinante, e, utilização de um adenovìrus

Country Status (15)

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

Families Citing this family (11)

* 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
CN1370240A (zh) * 1999-06-22 2002-09-18 阿文蒂斯药物股份有限公司 利用ppar核受体的表达调节系统
DE60115610T2 (de) * 2000-03-14 2006-10-19 Universite Pierre Et Marie Curie Durch Entzündungsvermittler induzierbare hybride Promotoren, diese enthaltende Vektoren und Verwendungen davon
FR2806418B1 (fr) 2000-03-14 2004-07-16 Aventis Pharma Sa Promoteurs hybrides inductibles par l'inflammation, vecteurs les contenant et utilisations
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
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
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
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0516443A1 (en) * 1991-05-31 1992-12-02 Eli Lilly And Company 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
ZA9710114B (en) 1998-06-08
HUP9904235A3 (en) 2003-03-28
CZ163399A3 (cs) 1999-08-11
HUP9904235A2 (hu) 2000-04-28
WO1998021349A1 (fr) 1998-05-22
FR2755699B1 (fr) 1998-12-18
EP0946740A1 (fr) 1999-10-06
AU5057898A (en) 1998-06-03
SK61499A3 (en) 2000-02-14
CA2269864A1 (fr) 1998-05-22
NO992153D0 (no) 1999-05-04
KR20000053163A (ko) 2000-08-25
NO992153L (no) 1999-05-04
JP2001503279A (ja) 2001-03-13
FR2755699A1 (fr) 1998-05-15
US20020144302A1 (en) 2002-10-03
IL129593A0 (en) 2000-02-29

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

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