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Using nanoparticles, in vivo gene therapy activates brain stem cells

brid, 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, explained Dhruba J. Bharali, Ph.D., a co-author on the paper and post-doctoral associate in the UB Department of Chemistry and UB's Institute for Lasers, Photonics and Biophotonics.

While they are easier and faster to produce, non-viral vectors typically suffer from very low expression and efficacy rates, especially in vivo.

"This is the first time that a non-viral vector has demonstrated efficacy in vivo at levels comparable to a viral vector," Bharali said.

In the UB experiments, targeted dopamine neurons -- which degenerate in Parkinson's disease, for example -- took up and expressed a fluorescent marker gene, demonstrating the ability of nanoparticle technology to deliver effectively genes to specific types of cells in the brain.

Using a new optical fiber in vivo imaging technique (CellviZio developed by Mauna Kea Technologies of Paris), the UB researchers were able to observe the brain cells expressing genes without having to sacrifice the animal. Then the UB researchers decided to go one step further, to see if they could not only observe, but also manipulate the behavior of brain cells.

Their finding that the nanoparticles successfully altered the development path of neural stem cells is especially intriguing because of scientific concerns that embryonic stem cells may not be able to function correctly since they have bypassed some of the developmental stages cells normally go through.

"What we did here instead was to reactivate adult stem cells located on the floor of brain ventricles, germinal cells that normally produce progeny that then die if they are not u
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Source:University at Buffalo


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