PhD studentship in Translational Research in Advanced Cell and Gene Therapies for Heart Failure

Mutations in the nuclear envelope are the second highest cause of familial dilated cardiomyopathy. We have identified novel mutations in the nuclear envelope in heart failure patients at the inherited cardiomyopathy clinic at Guy’s and St. Thomas’ Hospitals. However, the underlying molecular mechanisms that cause disease are poorly understood, which is due in part to a lack of appropriate human tissue models to study them.

This PhD project will use CRISPR/Cas9 technology to engineer these novel patient mutations combined state-of-the-art tissue engineering to generate ‘micro-hearts’ in a dish and light-sheet microscopy to reveal the functional effects of these mutations. Once established, the effects of clinically-approved or experimental drugs will be tested and the data relayed to geneticists and clinicians treating these patients.