Treatment of heart attacks is still limited since there are no available therapeutics capable of regeneration or repair of the heart cells.
We are using single‑cell technologies to map the DNA 'switches' that turn genes on in damaged heart cells. This will allow us to build precise tools to deliver treatment only where it is required. Testing these tools in advanced lab models will identify safe, effective therapies that repair heart muscle while limiting side effects.
Challenges
Cardiac deliveryOrganisations
- University of Edinburgh
In depth
Treatment of cardiovascular disease complications - such as myocardial infarction (MI) and heart failure (HF) - are to date still limited in terms of efficacy and specificity. There are currently no available therapeutics capable of selective and effective regeneration/ repair of injured cardiomyocytes and endothelial cells or recovering heart function. In fact, all current drug-based treatments only assist the surviving heart muscle and blood vessels after MI, but do not reverse disease progression.
To address that we aim to develop precision gene therapies for MI and HF by engineering cell‑ and disease‑state–specific cis‑regulatory modules (CRMs). Using numerous datasets, we will identify transcriptional and chromatin signatures of disease‑enriched cardiomyocyte and endothelial subtypes to design promoter–enhancer combinations with high specificity. CRM libraries will be assembled and screened in advanced cardiac in vitro models for activity and off‑target effects. Top candidates will be validated in our cardiac models. Lead CRMs will drive therapeutic RNA expression selectively in diseased cells to enhance repair while minimizing off‑target effects.