Rare Disease Report

Gene Therapy and Tyrosinemia Type 1

NOVEMBER 15, 2015
Joseph Lillegard, MD

Joseph Lillegard, MD, of the Mayo Clinic in Rochester, MN talks about the study they presented at the AASLD Annual Meeting (Liver Meeting) in which gene therapy was successfully used on a large animal model of hereditary tyrosinemia type 1 (HT-1).


Hickey RD, Elgilani F, Mao SA, et al. Autologous hepatocyte transplantation after ex vivo gene therapy in a large animal model of metabolic liver disease. Hepatol. 2015;62 (1 suppl): Abstract 2.

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