Dunpeng Cai
October 14, 2025
Contact: Brian Consiglio, consigliob@missouri.edu
Photo by Abbie Lankitus
The University of Missouri is breaking new ground in the treatment of genetic disorders, and it’s starting with Marfan syndrome — a potentially fatal genetic disorder that weakens the aorta, the heart’s main artery.
With a grant from the American Heart Association, Mizzou’s Dunpeng Cai is creating a novel RNA-based therapy to treat Marfan syndrome, which is caused by a mutation in the fibrillin-1 protein. Left untreated, it can lead to aortic aneurysms — tears or ruptures in the aorta — which can be deadly.
One current treatment, DNA editing, is risky because it can cause irreversible damage if a mistake is made. Cai’s approach is safer by targeting RNA instead.
“The goal of our new therapy is to correct the mutated RNA for those with Marfan syndrome, thus producing the fibrillin-1 protein in its correct form, ultimately curing this genetic disorder,” Cai, an assistant professor in the School of Medicine, said.
With the resources of the Roy Blunt NextGen Precision Health building at Mizzou, Cai is well-positioned to eventually take this research toward clinical trials. If successful, this RNA editing method could be applied to treat a wide range of genetic disorders, including Huntington’s disease, Down syndrome and sickle cell disease.
The RNA-based therapy may also have implications for cancer treatment. Some patients do not respond to current therapies due to specific genetic mutations. Cai’s system could one day correct those mutations, making treatments more effective.
Cai’s work is part of Mizzou’s broader push into precision medicine — tailoring treatments to the unique genetic makeup of individual patients. By sequencing a patient’s genes, Cai can pinpoint the exact location of the mutation and customize the RNA-based therapy accordingly.
“This innovative RNA-based approach could be a game-changer for precision medicine,” Cai said. “Mizzou is the perfect place for this research, as the Genomics Technology Core helps make this research successful.”
Cai’s interest in precision medicine began during his doctoral studies in pharmacology, when he questioned why traditional drugs worked well for some patients with genetic disorders but not for others. He discovered that while patients may share the same affected protein, the precise mutations can differ — a realization that inspired his current RNA-focused work.
Cai completed both his doctoral studies and postdoctoral training at Mizzou before becoming a faculty member. He credits Shiyou Chen and Stephen Barnes in the School of Medicine for their mentorship and encouragement.
“Searching for new ways to improve the quality of life for those with genetic disorders is what motivates my work,” Cai said. “Since those with Marfan syndrome have weaker connective tissue in their heart, bones, lungs and eyes, they get tired more easily and may not be able to exercise much or do physical activities they enjoy. Helping people is my current goal, and if this new approach works, Marfan syndrome is just the tip of the iceberg.”