<i>Ex Vivo</i> Hepatic Gene Therapy of a Mouse Model of Hereditary Tyrosinemia Type I

Abstract excerpt

Previously, this lab has reported the use of hepatocyte transplantation and in vivo gene therapy for the correction of a mouse model of Hereditary Tyrosinemia Type I (HT1). Here, we demonstrate repopulation of fumarylacetoacetate hydrolase (FAH)-deficient livers with cultured hepatocytes. Correction of the disease phenotype was achieved by retrovirally transducing cultured FAH¯ hepatocytes ex vivo, followed by...

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