Next-generation RNA therapeutics for cardiovascular disease

A heart attack kills heart muscle cells, and the body replaces them with stiff scar tissue that cannot pump blood—this project aims to stop that scarring by delivering synthetic genetic instructions directly to the heart. Heart attacks remain a leading cause of death and disability worldwide. After a heart attack, the heart’s ability to pump is permanently reduced because it cannot regenerate lost muscle. Current treatments reopen blocked arteries and manage symptoms, but none repair the damaged tissue itself. This research addresses that gap by using two types of RNA-based drugs: messenger RNA (mRNA) to instruct surviving heart cells to grow new blood vessels, and antisense oligonucleotides (ASOs) to block the molecular signals that trigger scarring. If successful, this work could produce the first-ever therapies that actively heal the heart after a heart attack, potentially preventing the downward spiral into heart failure that affects hundreds of thousands of people in the UK alone. The researcher is developing chemically modified RNA molecules that survive long enough in the bloodstream to reach heart cells, then testing them in preclinical models. This is still fundamental science—no human trials are imminent—but the underlying synthetic nucleic acid technology could eventually apply to other organs damaged by fibrosis, such as the liver or lungs.