Ex vivo hepatic gene therapy of a mouse model of hereditary tyrosinemia type I

Ken Overture, Muhsen Al-Dhalimy, Kara Manning, Ching Nan Ou, Milton Finegold, Markus Grompe

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Abstract

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 transplantation and selective repopulation. Treated mice were phenotypically normal and had corrected plasma amino acid levels and liver function tests. Our results demonstrate that efficient hepatic repopulation using ex vivo genetically manipulated hepatocytes is feasible.

Original languageEnglish (US)
Pages (from-to)295-304
Number of pages10
JournalHuman Gene Therapy
Volume9
Issue number3
Publication statusPublished - Feb 10 1998

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ASJC Scopus subject areas

  • Genetics

Cite this

Overture, K., Al-Dhalimy, M., Manning, K., Ou, C. N., Finegold, M., & Grompe, M. (1998). Ex vivo hepatic gene therapy of a mouse model of hereditary tyrosinemia type I. Human Gene Therapy, 9(3), 295-304.