Using Nanoparticles To Repair Brain Cells Damaged By Disease, Trauma Or Stroke

Using Nanoparticles, In Vivo Gene Therapy Activates Brain Stem Cells: Technique May Allow Scientists To Repair Brain Cells Damaged By Disease, Trauma Or Stroke

From: University at Buffalo

Using customized nanoparticles that they developed, University at Buffalo scientists have for the first time delivered genes into the brains of living mice with an efficiency that is similar to, or better than, viral vectors and with no observable toxic effect, according to a paper published this week in Proceedings of the National Academy of Sciences.

In addition to delivering therapeutic genes to repair malfunctioning brain cells, the nanoparticles also provide promising models for studying the genetic mechanisms of brain disease.

Viral vectors can be produced only by specialists under rigidly controlled laboratory conditions. By contrast, the nanoparticles developed by the UB team can be synthesized easily in a matter of days by an experienced chemist.

The UB researchers make their nanoparticles from hybrid, organically modified silica (ORMOSIL), the structure and composition of which allow for the development of an extensive library of tailored nanoparticles to target gene therapies for different tissues and cell types. A key advantage of the UB team's nanoparticle is its surface functionality, which allows it to be targeted to specific cells.

This is the first time that a non-viral vector has demonstrated efficacy in vivo at levels comparable to a viral vector. In the future, this technology may make it possible to repair neurological damage caused by disease, trauma or stroke.

Journal: Proc. Natl. Acad. Sci. USA, 10.1073/pnas.0504926102