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Article

Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model.

Journal of thrombosis and haemostasis : JTH - 1 Jun 2006

McCORMACK W M, Seiler M P, Bertin T K, Ubhayakar K, Palmer D J, Ng P, Nichols T C, Lee B

Abstract excerpt

BACKGROUND: Adenoviral vector-mediated gene therapy might have potential for long-term correction of the monogenic disease hemophilia A. OBJECTIVE: In this study, we tested the efficacy of administering a helper-dependent adenoviral vector (HDV) designed for maximal liver-restricted canine factor VIII (cFVIII) expression on three out-bred hemophilia A dogs. METHODS: Three FVIII-deficient animals from the...

Topics

Adenoviridae
Animals
Blood Coagulation
Disease Models, Animal
Dogs
Factor VIII
Genetic Therapy
Genetic Vectors
Hemophilia A
Liver
Mutation

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Partial Thromboplastin Time

Whole Blood Coagulation Time

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Identifiers

PMID: 16706963
DOI: 10.1111/j.1538-7836.2006.01901.x
PMCID: PMC3947717

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