Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease


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… (More)
DOI: 10.1038/gt.2011.186

8 Figures and Tables


  • Presentations referencing similar topics