The New Gene-Therapy Technique Has the Potential to Reverse Disease Processes

Scientists have developed a new gene-therapy method by transforming human cells into mass producers of tiny nano-sized particles filled with genetic materials that have the potential to reverse disease processes.

Although the research was supposed as evidence of concept, the experimental therapy slowed tumor growth and prolonged survival in mice with gliomas, which represent about 80 % of malignant brain tumors in people.

The approach takes benefit of exosomes, fluid-filled sacs that cells launch as a method to communicate with other cells.

Whereas exosomes are gaining ground as biologically friendly carriers of therapeutic supplies—as a result of there are lots of them, and so they do not prompt an immune response—the trick with gene therapy is discovering a technique to match these comparatively large genetic directions inside their tiny our bodies on a scale that may have a therapeutic impact.

This new technique depends on patented know-how that prompts donated human cells equivalent to adult stem cells to spit out millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug. When they’re injected into the bloodstream, they know precisely the place within the body to search out their target—even when it is within the brain.

By wanting further into the mechanism behind TNT’s success, scientists in Lee’s lab found that exosomes have been the key to delivering regenerative goods to tissue far under the skin’s surface.

The technology was adopted on this research into a way first writer Zhaogang Yang, a former Ohio State postdoctoral researcher now on the University of Texas Southwestern Medical Center, termed cellular nanoporation.