Research Summary

Innovative multi-model approach to identify novel candidate genes and small chemical molecules for cardiomyopathies

Cardiomyopathies (CM) are a group of life-threatening heart muscle diseases that represent a large proportion of pediatric heart failure and are one of the leading causes of heart transplantation in children. Every year about 1,000-5,000 new cases are diagnosed with pediatric cardiomyopathy. The cause of a significant percentage of childhood cardiomyopathies (~50%) remains unknown with no curative therapy available for these affected children. The long-term goal of this project is to identify genetic causes and curative drugs for childhood onset cardiomyopathies