University at Buffalo scientists have delivered genes into the brains of living mice with no observable toxic effect.

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.

The nanoparticles are constructed from hybrid, organically modified silica (ORMOSIL), which allowd for the development of an extensive library of tailored nanoparticles to target gene therapies for different tissues and cell types.