Tiny particle a big step in cancer gene therapy

Gene therapy is an attractive concept with great promise. But in practice, delivering therapeutic genes to diseased cells has been undermined by limited efficiency and considerable toxicity.

To overcome these hurdles, a group led by W. Mark Saltzman, Ph.D., chair and Goizueta Foundation Professor of Biomedical Engineering, Research Scientist Zhaozhong Jiang, Ph.D., and Associate Research Scientist Jiangbing Zhou, Ph.D., synthesized nanoparticles combining sticky DNA-binding regions with regions that protect the genetic material and ferry it safely into target cells.

As reported online in Nature Materials on December 4, when these biodegradable polymer-based particles were loaded with a gene that promotes cell death and injected into cancer-bearing mice, the growth of tumors in the mice was significantly inhibited, and they suffered no toxic side effects.

Saltzman hopes to someday use a nanoparticle delivery system in gene therapy for several human diseases, particularly brain tumors. “We’ve been developing techniques for introducing particles like these directly into the brain to treat malignant brain tumors for some years,” Saltzman says. “Our hope is that these polymers give us another tool—a very safe tool—that we can potentially use in that arena.”