The researchers, whose work is published in Science, demonstrated the new approach with an mRNA preparation that reprograms T cells—a powerful type of immune cell—to attack heart fibroblast cells.
“Fibrosis underlies many serious disorders, including heart failure, liver disease, and kidney failure, and this technology could turn out to be a scalable and affordable way to address an enormous medical burden,” says senior author Jonathan A. Epstein, chief scientific officer for Penn Medicine and executive vice dean and the William Wikoff Smith Professor of Cardiovascular Research in the Perelman School of Medicine. “But the most notable advancement is the ability to engineer T cells for a specific clinical application without having to take them out of the patient’s body.”
The new technique is based on chimeric antigen receptor (CAR) T cell technology, which, until now, has required the harvesting of a patient’s T cells and their genetic reprogramming in the lab to recognize markers on specific cell types in the body. These specially targeted T cells can then be multiplied using cell culture techniques and reinfused into the patient to attack a specific cell type.
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