Treating cardiac vascular dysfunction in Friedreich ataxia using human iPSC-derived multicellular cardiac organoids

Friedreich ataxia is an autosomal recessive neuromuscular disease and the most common inherited ataxia. Cardiomyopathy is the leading cause of premature death in Friedreich ataxia patients. Clinical reports indicate that the cardiomyopathy may be associated with abnormalities of the small coronary arteries. We have recently discovered multiple genes which may be involved in the disease pathogenesis. This project will knockdown key genes in the cardiac vascular system to determine their role in Friedreich ataxia cardiomyopathy using a cutting-edge 3D multicellular human cardiac organoid model. The knowledge and skills involved in this project are suitable for students who are interested in stem cell biology, metabolic and mitochondrial diseases, and heart disease. A student working on this project will have the opportunity to learn various experimental skills, including cell culture, protein and gene analysis, histology and electrophysiology.