The injection orders genetic instructions that stimulate muscle cells to produce a precursor to atrial natriuretic peptide (ANP), a hormone that promotes blood vessel growth.
An innovative experimental therapy using RNA technology could offer a new way of repair heart damage after a heart attack, addressing one of cardiology’s most persistent challenges: the heart’s limited ability to regenerate.
published in the journal Science, was led by Ke Cheng of Columbia Engineering. Researchers have developed a new approach that transforms the patient’s own body in a “medicine factory”producing healing molecules that become active only when they reach the heart.
Unlike traditional treatments, which often require invasive procedures to deliver medications directly to heart tissue, this method involves a simple injection into a muscle in the arm or thigh. The injected particles carry genetic instructions that stimulate muscle cells to produce an inactive precursor of an essential hormone. Once in the bloodstream, this molecule travels to the heart, where it is activated to promote repair.
At the heart of the therapy is atrial natriuretic peptide (ANP), a hormone known to promote blood vessel growthreduce inflammation and limit scar formation. Although newborn mammals naturally produce high levels of ANP, this ability decreases dramatically with age, says the .
To overcome this limitation, the team used RNA-lipid nanoparticles to provide instructions for producing the ANP precursor through the Nppa gene. Activation occurs specifically in the heart thanks to an enzyme called Corin, which is much more abundant in cardiac tissue than in other parts of the body.
In preclinical trials involving small and large animals, a single injection significantly reduced scar tissue and improved heart function. The therapy has also proven effective in challenging scenarios, including elderly individuals, animals with diabetes and cases where treatment was delayed until a week after the heart attack.
To prolong its effects, the researchers used self-amplifying RNA, allowing the treatment to remain active up to four weeks after a single dose. This can reduce the need for frequent hospital visits, making therapy more practical for patients.
The cardiologists involved in the study highlight the potential impact. Although current treatments can restore blood flow after a heart attack, do little to reverse permanent loss of cardiac muscle cells, which often leads to heart failure over time.
The research team plans to advance to human trials, with initial trials planned for Columbia University Irving Medical Center.
