Penn Medicine research teams are using science to develop a new treatment for heart disease that could potentially work with just one injection – three different ways a single injection could one day cure the heart. continues to advance.
Image: iStock/Prostock-Studio
Kiran Musunur, a cardiologist at Pennsylvania Medicine and director of the Genetic and Epigenetic Origins of Disease Program at Penn State Perelman School of Medicine, discovered the gene that regulates LDL cholesterol and identified proteins associated with that genetic pathway. This has inspired the development of multiple targeted drugs. His lab then developed a process that uses CRISPR gene editing technology to modify liver genes to permanently lower cholesterol levels and prevent heart attacks and strokes. The approach, a one-time, vaccine-like shot that, if successful, could prevent heart disease, is currently in clinical trials in the United Kingdom and New Zealand, has been approved by the U.S. Food and Drug Administration, and has been approved by the U.S. But we are planning to start testing soon. .
Nobel Prize-winning scientist Drew Wiseman is the Roberts Family Professor of Vaccine Research and director of the Penn RNA Innovation Institute, working on multiple uses of mRNA for the treatment and prevention of heart disease.
Working with Vlad Muzhikantov, a founding professor of nanoparticle research, Wiseman’s lab developed a method to inject mRNA to specifically affect heart cells. “Heart medicines are not heart-specific,” Wiseman says. “And when you’re trying to treat myocardial infarction or cardiomyopathy or other genetic defects in the heart, it’s very difficult because you can’t treat the heart.”
Penn Medicine researchers are also combining two of the biggest innovations: CAR T-cell therapy and mRNA therapy in an approach to treating fibrosis, a heart dysfunction that often causes heart failure.
For more information, visit Penn Medicine News.
