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Using nanoparticles, in vivo gene therapy activates brain stem cells

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.

The paper describes how the UB scientists used gene-nanoparticle complexes to activate adult brain stem/progenitor cells in vivo, demonstrating that it may be possible to "turn on" these otherwise idle cells as effective replacements for those destroyed by neurodegenerative diseases, such as Parkinson's.

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.

"Until now, no non-viral technique has proven to be as effective as the viral vectors in vivo," said co-author Paras N. Prasad, Ph.D., executive director of the UB Institute for Lasers, Photonics and Biophotonics, SUNY Distinguished Professor in UB's Department of Chemistry and principal investigator of the institute's nanomedicine program. "This transition, from in vitro to in vivo, represents a dramatic leap forward in developing experimental, non-viral techniques to study brain biology and new therapies to address some of the most debilitating human diseases."

Viral vectors for gene therapy always carry with them the potential to revert back to wild-type, and some human trials have even resulted in fatalities.

As a result, new research focuses increasingly on non-viral vectors, which don't carry this risk.

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 hy
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Source:University at Buffalo


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