Nanoparticles can be used in gene therapy to transfer therapeutic genes in to brain cells and helps in studying genetic mechanism of brain diseases.
Gene-nanoparticle complexes developed by scientists at the University of Buffalo, has proved to deliver therapeutic genes directly into the brains of living mice, the transfer rate is comparatively higher to transfer rates of viral vectors and the procedure does not cause any side effects. The result of this work is published in this week Proceedings of the National Academy of Sciences (PNAS). This procedure is easier and faster in transferring desired genes compared to non-viral vectors which has very low expression and efficacy rates, especially in vivo. This gene nanoparticle complex also helps to study the genetic mechanism of brain diseases in addition to offering repair mechanism.
The scientists used this technique 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.
"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. 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 and the therapy can be targeted specifically against the desired cells "This is the first time that a non-viral vector has demonstrated efficacy in vivo at levels comparable to a viral vector," Bharali said.
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. Using this technique it is also possible to manipulate the behaviour of the brain cells.
Related Information on Stem cells in Medindia:
Information on stem cells - Stem cells are used to treat degenerative diseases, which occur due to premature cell death or malfunctioning of specific cell type. Stem cells can be used to replace these dead cells.
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