We have developed a new technology to reprogram human cells to go back to a state from which they can then develop into other kinds of cells. Our technology is based on a strain of Measles virus, one of the safest vaccines in human history. In this project we will use modify our technology to correct genetic mutation in the human genome and particularly in patients with inherited red blood cell disorders, such as sickle cell disease.

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: