WO1998046781A3 - Novel transgene expression system for increased persistence - Google Patents

Novel transgene expression system for increased persistence Download PDF

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Publication number
WO1998046781A3
WO1998046781A3 PCT/US1998/007841 US9807841W WO9846781A3 WO 1998046781 A3 WO1998046781 A3 WO 1998046781A3 US 9807841 W US9807841 W US 9807841W WO 9846781 A3 WO9846781 A3 WO 9846781A3
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WO
WIPO (PCT)
Prior art keywords
expression system
transgene expression
transgene
directed
compositions
Prior art date
Application number
PCT/US1998/007841
Other languages
French (fr)
Other versions
WO1998046781A2 (en
Inventor
Donna Armentano
John Marshall
Nelson Yew
Seng Cheng
Richard J Gregory
Original Assignee
Genzyme Corp
Donna Armentano
John Marshall
Nelson Yew
Seng Cheng
Richard J Gregory
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 Genzyme Corp, Donna Armentano, John Marshall, Nelson Yew, Seng Cheng, Richard J Gregory filed Critical Genzyme Corp
Priority to EP98918409A priority Critical patent/EP0975786A2/en
Priority to CA002287076A priority patent/CA2287076A1/en
Priority to JP54434398A priority patent/JP2001521390A/en
Priority to AU71336/98A priority patent/AU7133698A/en
Publication of WO1998046781A2 publication Critical patent/WO1998046781A2/en
Publication of WO1998046781A3 publication Critical patent/WO1998046781A3/en

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • 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
    • 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
    • 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
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pulmonology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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

The invention is directed to a transgene expression system comprising a transcription unit which contains a transgene operably linked to expression control sequences, preferably the CMV promoter, and which is delivered simultaneously with all or part of the adenovirus E4 genomic region to a cell in order to facilitate persistent expression of the transgene. The components of the transgene expression system can be delivered by vectors including plasmids and/or viruses and may be complexed with cationic amphiphiles to facilitate entry into a cell. The invention is also directed to methods for the production of the transgene expression system. The invention is further directed to compositions that contain the transgene expression system and to methods for the use of such compositions to deliver transgenes encoding biologically active proteins to cells.
PCT/US1998/007841 1997-04-14 1998-04-14 Novel transgene expression system for increased persistence WO1998046781A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP98918409A EP0975786A2 (en) 1997-04-14 1998-04-14 Novel transgene expression system for increased persistence
CA002287076A CA2287076A1 (en) 1997-04-14 1998-04-14 Novel transgene expression system for increased persistence
JP54434398A JP2001521390A (en) 1997-04-14 1998-04-14 Novel transgene expression system for enhanced persistence
AU71336/98A AU7133698A (en) 1997-04-14 1998-04-14 Novel transgene expression system for increased persistence

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/839,552 1997-04-14
US08/839,552 US5981275A (en) 1997-04-14 1997-04-14 Transgene expression system for increased persistence

Publications (2)

Publication Number Publication Date
WO1998046781A2 WO1998046781A2 (en) 1998-10-22
WO1998046781A3 true WO1998046781A3 (en) 1999-01-21

Family

ID=25280040

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/007841 WO1998046781A2 (en) 1997-04-14 1998-04-14 Novel transgene expression system for increased persistence

Country Status (6)

Country Link
US (1) US5981275A (en)
EP (1) EP0975786A2 (en)
JP (1) JP2001521390A (en)
AU (1) AU7133698A (en)
CA (1) CA2287076A1 (en)
WO (1) WO1998046781A2 (en)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2705686B1 (en) 1993-05-28 1995-08-18 Transgene Sa New defective adenoviruses and corresponding complementation lines.
DE69633565T3 (en) 1995-06-15 2013-01-17 Crucell Holland B.V. PACKAGING SYSTEMS FOR HUMAN, HUMAN ADENOVIRES, FOR USE IN GENE THERAPY
IL135578A0 (en) * 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
AU750803B2 (en) 1997-10-29 2002-07-25 University Of Pittsburgh Use of vectors such as adenoviruses and/or adeno associated viruses and/or retroviruses and/or herpes simplex viruses and/or liposomes and/or plasmids as a vehicle for genetic information enabling mammal cells to produce agents for the treatment of bone pathologies
WO1999036557A1 (en) * 1998-01-16 1999-07-22 Genzyme Corporation Novel promoter elements for persistent gene expression
EP0974668B1 (en) * 1998-07-07 2002-10-02 Transgene S.A. Use of adenoviral E4 reading frames to improve expression of a gene of interest
WO2000012740A2 (en) 1998-08-28 2000-03-09 Duke University ADENOVIRUSES DELETED IN THE IVa2, 100K AND/OR PRETERMINAL PROTEIN SEQUENCES
US6440944B2 (en) * 1998-10-16 2002-08-27 Genvec, Inc. Methods of administering adenoviral vectors
US6929946B1 (en) * 1998-11-20 2005-08-16 Crucell Holland B.V. Gene delivery vectors provided with a tissue tropism for smooth muscle cells, and/or endothelial cells
DE19900284B4 (en) * 1999-01-07 2008-02-07 Ingo Prof. Dr. Schmidt-Wolf Method for the efficient introduction of DNA into eukaryotic cells using an adenoviral vector
ATE431421T1 (en) * 1999-01-28 2009-05-15 Onyx Pharma Inc ADENOVIRAL SHUTTLE VECTORS DELETED IN THE E1B REGION
US6764674B1 (en) 1999-01-28 2004-07-20 Onyx Pharmaceuticals Inc. Adenovirus E1B shuttle vectors
US6913922B1 (en) * 1999-05-18 2005-07-05 Crucell Holland B.V. Serotype of adenovirus and uses thereof
US7468181B2 (en) * 2002-04-25 2008-12-23 Crucell Holland B.V. Means and methods for the production of adenovirus vectors
AU2001259174A1 (en) * 2000-04-28 2001-11-12 University Of Pittsburgh Of The Commonwealth System Of Higher Education Viral and non-viral vectors as vehicles for delivering transgenes for treating bone pathologies
US6649597B1 (en) 2000-06-22 2003-11-18 The University Of Iowa Research Foundation Targeting vector to the urokinase plasminogen activator receptor
US6579522B1 (en) 2000-06-27 2003-06-17 Genvec, Inc. Replication deficient adenoviral TNF vector
US7198924B2 (en) 2000-12-11 2007-04-03 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
JP2004516016A (en) * 2000-09-18 2004-06-03 ジェンザイム・コーポレイション Expression vector containing hybrid ubiquitin promoter
US20030086903A1 (en) 2001-11-02 2003-05-08 Genvec, Inc. Therapeutic regimen for treating cancer
EP1327688A1 (en) 2002-01-14 2003-07-16 Vereniging Voor Christelijk Wetenschappelijk Onderwijs Adenoviruses with enhanced lytic potency
JP2005529959A (en) * 2002-06-14 2005-10-06 マイラス コーポレイション Novel method for delivering polynucleotides to cells
EP2287341B1 (en) 2003-12-01 2013-02-13 Life Technologies Corporation Nucleic acid molecules containing recombination sites and methods of using the same

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994028938A1 (en) * 1993-06-07 1994-12-22 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy sponsorship
WO1996013596A1 (en) * 1994-10-28 1996-05-09 Rhone-Poulenc Rorer S.A. Viable contaminant particle free adenoviruses, their preparation and use
WO1996018372A2 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
WO1996030534A1 (en) * 1995-03-24 1996-10-03 Genzyme Corporation Adenovirus vectors for gene therapy

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US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5635380A (en) * 1994-01-18 1997-06-03 Vanderbilt University Enhancement of nucleic acid transfer by coupling virus to nucleic acid via lipids
US5719131A (en) * 1994-12-09 1998-02-17 Genzyme Corporation Cationic amphiphiles containing dialkylamine lipophilic groups for intracellular delivery of therapeutic molecules
CA2625279A1 (en) * 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav

Patent Citations (4)

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WO1994028938A1 (en) * 1993-06-07 1994-12-22 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy sponsorship
WO1996013596A1 (en) * 1994-10-28 1996-05-09 Rhone-Poulenc Rorer S.A. Viable contaminant particle free adenoviruses, their preparation and use
WO1996018372A2 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
WO1996030534A1 (en) * 1995-03-24 1996-10-03 Genzyme Corporation Adenovirus vectors for gene therapy

Non-Patent Citations (1)

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Title
BRIDGE, E. ET AL.: "Adenovirus early region 4 and viral DNA synthesis", VIROLOGY, vol. 193, 1993, pages 794 - 801, XP002051623 *

Also Published As

Publication number Publication date
AU7133698A (en) 1998-11-11
CA2287076A1 (en) 1998-10-22
JP2001521390A (en) 2001-11-06
WO1998046781A2 (en) 1998-10-22
EP0975786A2 (en) 2000-02-02
US5981275A (en) 1999-11-09

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