WO2001018224A8 - Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia - Google Patents

Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia

Info

Publication number
WO2001018224A8
WO2001018224A8 PCT/US2000/023692 US0023692W WO0118224A8 WO 2001018224 A8 WO2001018224 A8 WO 2001018224A8 US 0023692 W US0023692 W US 0023692W WO 0118224 A8 WO0118224 A8 WO 0118224A8
Authority
WO
WIPO (PCT)
Prior art keywords
cystic fibrosis
fibrosis transmembrane
conductance regulator
transmembrane conductance
adenoviral vectors
Prior art date
Application number
PCT/US2000/023692
Other languages
French (fr)
Other versions
WO2001018224A1 (en
Inventor
Helen Romanczuk
Samuel C Wadsworth
Richard J Gregory
Original Assignee
Genzyme Corp
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 filed Critical Genzyme Corp
Priority to AU70855/00A priority Critical patent/AU7085500A/en
Publication of WO2001018224A1 publication Critical patent/WO2001018224A1/en
Publication of WO2001018224A8 publication Critical patent/WO2001018224A8/en

Links

Classifications

    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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

Abstract

Disclosed are recombinant viral vectors for gene transfer to the epithelium of patients. Preferred embodiments include adenoviral vectors designed for the efficient transfer of the DNA encoding the cystic fibrosis transmembrane conductance regulator (CFTR) to human airways in vivo. The present invention provides vectors for the enhanced expression and persistence of a gene such as CFTR, which may allow lower vector doses and/or longer intervals between readministration and which comprise, in operable linkage, the keratin 18 (K18) promoter/enhanier element, an intron, the transgene and a stability enhancing element.
PCT/US2000/023692 1999-09-08 2000-08-29 Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia WO2001018224A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU70855/00A AU7085500A (en) 1999-09-08 2000-08-29 Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15305699P 1999-09-08 1999-09-08
US60/153,056 1999-09-08

Publications (2)

Publication Number Publication Date
WO2001018224A1 WO2001018224A1 (en) 2001-03-15
WO2001018224A8 true WO2001018224A8 (en) 2001-07-05

Family

ID=22545601

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/023692 WO2001018224A1 (en) 1999-09-08 2000-08-29 Adenoviral vectors modified for increased and persistent expression of the cystic fibrosis transmembrane conductance regulator gene in human airway epithelia

Country Status (2)

Country Link
AU (1) AU7085500A (en)
WO (1) WO2001018224A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004504035A (en) * 2000-07-14 2004-02-12 ユニバーシティ　オブ　サスカチュワン Adenovirus vector containing intron

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU715543B2 (en) * 1995-09-08 2000-02-03 Genzyme Corporation Improved AAV vectors for gene therapy
JP2002508967A (en) * 1998-01-16 2002-03-26 ジェンザイム・コーポレイション Adenovirus vector with modified capsid protein

Also Published As

Publication number Publication date
AU7085500A (en) 2001-04-10
WO2001018224A1 (en) 2001-03-15

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