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Using human stem cells to understand inherited heart diseases

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Using human stem cells to understand inherited heart diseases

Dr James McNamara, Murdoch Children's Research Institute

2023 Future Leader Fellowship - Level 1

Years funded: 2024 - 2027

Genetic cardiomyopathy is a form of heart disease that causes abnormal growth and pumping function of the heart and affects up to 35 million people globally. With limited treatment options and an annual cost of over $3 billion per year in Australia alone, the need for new therapies for these patients cannot be overstated. My program builds on my 10 years of pioneering research into inherited cardiomyopathy and uses human pluripotent stem cell models to understand the molecular causes of inherited cardiomyopathy. My long-term vision is to develop a research program using innovative approaches for the identification of novel therapeutic targets for inherited cardiomyopathy. A key goal of this Future Leader Fellowship is to define how a novel signalling pathway that I discovered controls heart function using cutting-edge human pluripotent stem cell models and in-depth molecular profiling. By generating a deep understanding of this pathway, I will uncover new molecular targets that may improve heart function in inherited cardiomyopathy.

Last updated12 March 2024