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Genetic correction of variant alleles as a treatment for inherited cardiomyopathies

  • 1 July 2021 to 31 December 2021
  • Awards: Pump-priming Awards

Cardiomyopathies – including hypertrophic (HCM), dilated (DCM) and arrhythmogenic right ventricular (ARVC) – are amongst the most common severe inherited diseases, with a combined prevalence of around 1 in 250. The genetic cause and primary triggers of these diseases are now quite well understood – cardiomyopathies are predominantly caused by mutations in patients DNA that alter the ability of the heart muscle to beat.

Current therapies are aimed at reducing symptoms rather than correcting the genetic cause of the disease. With this award we are now embarking on studies to test specific strategies of gene correction in patient derived induced pluripotent stem cells.