Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A.

doi:10.1182/blood-2003-05-1426

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Article

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A.

Blood - 1 Feb 2004

Brown Brian D, Shi Chang Xin, Powell Sandra, Hurlbut David, Graham Frank L, Lillicrap David

Abstract excerpt

Two helper-dependent (HD) adenoviral vectors encoding a canine factor VIII B-domain-deleted transgene (cFVIII) were constructed and evaluated in 4 hemophilia A dogs. One vector was regulated by the cytomegalovirus (CMV) promoter (HD-CMV-cFVIII), while the other vector contained a tissue-restricted promoter comprised of the human FVIII proximal promoter with an upstream concatemer of 5 hepatocyte nuclear factor 1...

Topics

Acute-Phase Reaction
Adenoviridae
Animals
Base Sequence
DNA, Recombinant
Disease Models, Animal
Dogs
Factor VIII
Gene Expression
Genetic Therapy
Genetic Vectors

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Helper Viruses

Hemophilia A

Liver

Phenotype

Promoter Regions, Genetic

RNA

Recombinant Proteins

Source

Open in PubMed

Identifiers

PMID: 14512318
DOI: 10.1182/blood-2003-05-1426

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