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

ABSTRACT:

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 University of North Carolina colony were injected with 1 x 10(12) (Dog A), and 3 x 10(12) (Dog B and C) vp kg(-1) helper-dependent adenoviral vector, and we performed systematic analysis of toxicity, persistence of therapeutic gene expression, and molecular analysis of gene transfer.

Results

We observed acute dose-dependent elevation in liver enzymes and thrombocy

SUBMITTER: McCORMACK WM 

PROVIDER: S-EPMC3947717 | biostudies-literature | 2006 Jun

REPOSITORIES: biostudies-literature