Gene therapy has been around as a promising new treatment for a while now, however it is plagued with safety concerns because of the use of viral vectors, and a lack of efficiency with non-viral vectors. Researchers at the Johns Hopkins University School of Medicine have developed a technique that delivers gene therapy into human brain cancer (glioblastoma) cells using non-viral nanoparticles that can be freeze-dried and stored for up to three months prior to use.
The team tested various commercially available DNA-binding polymers, identifying polymers efficient in delivery to glioblastoma cells but also in healthy cells, as well as polymers that affect only glioblastoma cells and brain tumor stem cells without affecting healthy cells. A second advantage of these poly(beta-amino ester) nanoparticles is that they can be stored at 4 degrees Celsius for at least three months without losing efficiency or increasing toxicity, making them more practical in a clinical setting. There is still a long way to go before we will see any such treatment used in-vivo, but it is a promising step nonetheless. The results are published in the August issue of Biomaterials.
Press release: Just add water and… treat brain cancer…
Article abstract: Non-viral gene delivery nanoparticles based on Poly(?-amino esters) for treatment of glioblastoma…