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

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.

Year 1 Progress Report:

We successfully applied our first year of funding toward demonstrating safety and efficacy of our gene therapy in multiple animal models and investigating integration of the lentiviral vector at the genomic level. We have shown that genomic integration is benign, and that this treatment has not caused cancer in any of our short or long-term studies in any model. Next, we will bring the current data to the FDA for consideration of human patient trial application.