The nanoparticles, designed by researchers at the University at Buffalo, activate adult brain stem cells, suggesting that it could be possible to turn on the otherwise dormant cells to provide replacements for cells destroyed by neurodegenerative diseases such as Parkinson's.
The nanoparticles used in the study can be synthesized easily in days.
They are created from hybrid, organically modified silica (ORMOSIL), which allows for the development of a library of tailored nanoparticles to target gene therapies for different tissues and cell types.
Nonviral vectors typically suffer from low expression and efficacy rates, especially in vivo, but the researchers say that this study is the first time a nonviral vector has shown efficacy in vivo at levels comparable to a viral vector.
In the study, targeted dopamine neurons took up and expressed a fluorescent marker gene (see image), showing that the nanoparticle technology can effectively deliver genes to specific types of brain cells.
"In the future, this technology may make it possible to repair neurological damage caused by disease, trauma or stroke," says study coauthor Earl J. Bergey.
The team next plans to conduct similar studies in larger animals.
The research is reported in the Proceedings of the National Academy of Sciences.