Researchers at Mayo Clinic have developed a new targeted gene therapy approach for cholangiocarcinoma, a rare and aggressive bile duct cancer. The study, published in JHEP Reports, introduces a precision method that delivers treatment directly to cancer cells while limiting damage to healthy tissue.
The system uses small interfering RNA (siRNA) to switch off genes that drive tumor growth. These molecules are carried through the body by tiny fat-based nanoparticles derived from milk, which act as safe delivery vehicles. To ensure accuracy, researchers added a targeting component called an aptamer—a DNA strand identified after screening hundreds of trillions of candidates—that binds specifically to cholangiocarcinoma cells.
In preclinical testing, the therapy successfully reached cancer cells, reduced tumor growth, and increased cancer cell death. Importantly, it avoided harming nearby healthy tissue, suggesting fewer side effects than conventional treatments.
The technology is still in the preclinical stage, but it has already been patented. Researchers are now working to refine gene targets and develop personalized versions of the therapy based on each patient’s tumor profile.