Biomedical engineers at the USC Viterbi School of Engineering have developed a new gene therapy using naturally derived nanoparticles, offering a potential breakthrough in treating cardiovascular and kidney diseases. These two conditions continue to pose major public health challenges worldwide. In the U.S., the Centers for Disease Control and Prevention reports that more than one in seven adults suffer from chronic kidney disease, while cardiovascular disease remains the top cause of death.
Led by Dr. Eun Ji Chung, the research team has used a type of naturally occurring nanoparticle known as extracellular vesicles (EVs), which are secreted by human cells, to create a powerful therapeutic platform. Unlike synthetic nanoparticles, these natural vesicles are genetically engineered to deliver gene therapies with greater safety and efficiency.
The findings were published in two leading journals: Advanced Healthcare Materials for the cardiovascular research and Biomaterials for the kidney-related study.
Dr. Chung, who holds the Dr. Karl Jacob Jr. and Karl Jacob III Early Career Chair and is an assistant professor of biomedical engineering, chemical engineering, and materials science, has long studied drug delivery and nanomedicine. Her lab has previously focused on self-assembling synthetic nanoparticles for targeted drug treatments in diseases such as cancer and heart and kidney conditions. The current research expands that focus to naturally derived particles for enhanced biocompatibility and effectiveness.
“These natural nanoparticles, extracellular vesicles, are already produced by our cells,” Chung explained. “We modified the cells to secrete them in a more therapeutic form, increasing their potential to treat disease.”
The lab’s approach harnesses EVs released by healthy cells, which naturally carry RNA, proteins, and other helpful particles.
Although these naturally secreted EVs can reduce disease-related inflammation, their effects are typically weak. To address this, the team genetically engineered cells to produce EVs with elevated therapeutic qualities.
“In chronic conditions like atherosclerosis, we wanted a safer long-term therapy,” Chung said. “Instead of giving patients synthetic particles for life, we use natural particles that the body is already familiar with.”
A key innovation in the study involves enhancing microRNA 145 levels in the EVs. This particular microRNA targets vascular smooth muscle cells, which play a role in forming plaque inside arteries. By boosting its levels, the EVs are better equipped to prevent or slow atherosclerosis.
In direct comparison studies, Chung’s lab found that their engineered EVs were significantly more effective at reducing arterial plaque than synthetic nanoparticles. “We used matching doses and saw that EVs had better potency,” Chung noted. “Not only are they natural, but their properties allow them to enter cells more efficiently.”
This discovery opens up the potential for long-term, safer gene therapies for patients with chronic conditions like cardiovascular and kidney disease. The research team believes that natural EVs could form the foundation of next-generation precision medicine strategies.
As the work progresses, Chung and her colleagues aim to explore additional applications for their EV platform, which could extend to treating other chronic diseases. The findings underscore a growing interest in natural therapies that align with the body’s own systems to deliver lasting health benefits.
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