Year 1

The goal of our project was to use stem cells to help identify new drugs for the treatment of Danon Disease, a rare, inherited disease that causes severe heart disease. Patients with Danon disease generally die in the second and third decade of life of heart failure. We have been working on this project for roughly one year. Since starting we have developed multiple stem cell lines from patients with Danon Disease. We have used these stem cells to make heart cells and have begun testing medicines on these heart cells to see if we can get them to work better. We plan in the future to identify new medicines to test any new medicines we identify on mice that have been made to mimic the disease. We are very hopeful that by the end of this project we will have come up with new ways for helping patients with this deadly disease.