New Therapy Sparks Heart Regeneration in Lab Study

Modified RNA Approach Aims to Repair Damage from Heart Attacks

A groundbreaking therapy using modified messenger RNA (modRNA) has shown promise in regenerating heart tissue after simulated heart attacks in mice. Researchers have successfully reactivated a dormant gene, potentially unlocking the heart’s own repair mechanisms.

Unlocking Dormant Repair Genes

Heart attacks remain a leading cause of death and disability worldwide. The devastating reality is that heart muscle cells, called cardiomyocytes, do not regenerate after oxygen deprivation. Current medical interventions manage symptoms but cannot restore lost cardiac tissue. However, scientists at the Lewis Katz School of Medicine at Temple University have demonstrated that delivering the PSAT1 gene to damaged heart tissue in mice can stimulate repair and improve cardiac function.

Raj Kishore, the study’s corresponding author, explained the motivation behind the research: PSAT1 is a gene that is highly expressed during early development but becomes virtually silent in the adult heart. We wanted to explore whether reactivating this gene in adult heart tissue could promote regeneration after injury.

Promising Preclinical Results

The team utilized modRNA to introduce PSAT1 to mouse hearts immediately following a heart attack. This intervention led to increased proliferation of cardiomyocytes and new blood vessel formation. Significantly, the treated hearts exhibited reduced tissue scarring, resulting in markedly improved heart function and survival rates compared to control groups.

The therapy works by activating the serine synthesis pathway, a crucial metabolic network that supports cell division and resilience to stress. This metabolic boost creates an environment conducive to heart muscle cell survival and multiplication, enabling the formation of new, functional tissue instead of scar tissue.

Advantages of modRNA Technology

The use of modRNA offers distinct advantages. It allows for precise delivery of genes like PSAT1 with minimal side effects. Unlike gene therapies that employ viral vectors, modRNA does not integrate into the host’s genome, mitigating concerns about long-term genetic risks.

Globally, cardiovascular diseases result in an estimated 17.9 million deaths annually, according to the World Health Organization (WHO) (WHO 2023). This new therapeutic avenue offers hope for millions affected by heart damage.

Future Directions

Although still in the preclinical phase, the researchers are optimistic about the therapy’s potential. The next steps involve conducting further testing in larger animal models and refining delivery techniques in preparation for eventual human clinical trials.