Article
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia.
Blood - 1 Oct 2003
Ehrhardt Anja, Xu Hui, Dillow Aaron M, Bellinger Dwight A, Nichols Timothy C, Kay Mark A
Abstract excerpt
Many approaches for treating hemophilia via gene transfer have been attempted in large animal models but all have potential drawbacks. Recombinant adenoviral vectors offer high-efficiency transfer of an episomal vector but have been plagued by the cytotoxicity/immunogenicity of early-generation vectors that contain viral genes. In our current study, we have used a nonintegrating helper-dependent (HD) adenoviral...
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