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Gene therapy of hereditary Tyrosinemia Type I using in vivo lentiviral vectors

Research Grant Recipient: Joseph Lillegard, MD, PhD

Award Value: $249,998

Research Focus: Hereditary tyrosinemia

Project Summary: Hereditary Tyrosinemia Type 1 (HT1) is a genetic disorder that can lead to liver and kidney failure, and is fatal without treatment. There is no cure. This project aims to develop a lentivirus-mediated gene therapy to treat multiple inborn errors of liver metabolism, with the research in HT1 serving as a model for future studies of other rare liver diseases.