Large-Scale Generation of Functional mRNA-Encapsulating Exosomes Achieved by Nanoporation; Advance May Enable New Gene Therapy Strategies; Initial Study Done on Gliomas

Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes. Though the research was intended as a proof of concept, the experimental therapy slowed tumor growth and prolonged survival in mice with gliomas, which constitute about 80 percent of malignant brain tumors in humans. The technique takes advantage of exosomes, fluid-filled subcellular vesicles that cells release as a way to communicate with other cells. While exosomes are gaining ground as biologically friendly carriers of therapeutic materials – because there are a lot of them and they don’t elicit an immune response – the trick with gene therapy is finding a way to fit those comparatively large genetic instructions inside the tiny exosomes on a scale that will have a therapeutic effect. This new method relies on patented technology that prompts donated human cells such as adult stem cells to release millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug. When they are injected into the bloodstream, these exosomes know exactly where in the body to find their target – even if it’s in the brain. “Think of them like Christmas gifts: The gift is inside a wrapped container that is postage-paid and ready to go,” said senior study author L. James Lee, Phd, Professor Emeritus of Chemical and Biomolecular Engineering at The Ohio State University. And they are gifts that keep on giving, Dr. Lee noted: “This is a Mother Nature-induced therapeutic nanoparticle.” The new study was published online on December 16, 2019 in Nature Biomedical Engineering. The article is titled “Large-Scale Generation of Functional mRNA-Encapsulating Exosomes Via Cellular Nanoporation.”
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