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.