Article
Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease.
Gene therapy - 1 Nov 2012
Roybal J L, Endo M, Radu A, Gray L, Todorow C A, Zoltick P W, Lutsenko S, Flake A W
Abstract excerpt
The ideal gene therapy for metabolical liver disorders would target hepatocytes before the onset of disease and be durable, non-toxic and non-immunogenic. Early gestational gene transfer can achieve such goals. Here, we demonstrate that prenatal gene transfer of human Atp7b reduces liver pathology and improves biochemical markers in Atp7b(-/-) mice, a murine model of Wilson's disease (WD). Following prenatal...
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