Could a single shot heal heart disease?

Experts at Penn Medicine are researching novel treatments for heart disease, including CRISPR gene editing technology, CAR T technology, and mRNA injections.

Multiple research teams at Penn Medicine are continuing to advance the science to develop new treatments for heart disease that could work with just one shot—three different ways that a single injection could someday heal the heart.

A medical professional takes the blood pressure of a mature patient.
Image: iStock/Prostock-Studio

Penn Medicine cardiologist Kiran Musunuru, director of the Genetic and Epigenetic Origins of Disease Program in Penn’s Perelman School of Medicine, Musunuru discovered a gene that regulates LDL cholesterol, which inspired the development of multiple drugs targeting a protein related to that gene pathway. In his lab, he has since developed processes to use CRISPR gene editing technology to modify genes in the liver to permanently reduce cholesterol levels and therefore provide protection against heart attack and stroke. The approach, a one-time injection like a vaccine that might prevent heart disease if it is successful, is now in clinical trials in the U.K. and New Zealand and has been approved by the U.S. Food and Drug Administration to begin trials in the U.S. soon.

Nobel Prizewinning scientist Drew Weissman, the Roberts Family Professor in Vaccine Research and director of the Penn Institute for RNA Innovation, is working on multiple uses of mRNA to treat or prevent heart disease.

Working with Vlad Muzykantov, the Founders Professor in Nanoparticle Research, Weissman’s lab has developed a way to target an mRNA injection to act specifically in heart cells. “Drugs for heart disease aren’t specific for the heart,” Weissman says. “And when you’re trying to treat a myocardial infarction or cardiomyopathy or other genetic deficiencies in the heart, it’s very difficult, because you can’t deliver to the heart.”

Penn Medicine researchers are also combining two of their biggest innovations—CAR T cell therapy and mRNA therapeutics—as an approach to treat fibrosis, an impairment of heart function which is often a factor in heart failure.

Read more at Penn Medicine News.