Developing a gene therapy for heart disease

About the project:

Many heart and skeletal muscle diseases are caused by when the muscle contraction machinery is damaged. In the case of heart muscle, a common result of compromised muscle contraction is a condition called cardiomyopathy.

This study proposes to develop a gene therapy based on a gene called ALPK3 that improves the function of the contraction machinery in a patient’s heart muscle cells, thereby hoping to treat cardiomyopathy.

Lead researcher: A/Prof David Elliott,

Investigators: Prof Enzo Porrello, Dr James McNamara, A/Prof Richard Mills

Tissue of interest: Heart and skeletal muscles

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Proudly in partnership with

The Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, is supported by a Novo Nordisk Foundation grant number NNF21CC0073729​​