ABSTRACT
Nanotechnology has immense potential in the delivery of various therapeutic molecules into the brain for treating brain-speciϐic disorders. The brain is particularly amenable to gene therapy, where an exogenously administered transgene can stimulate neuronal stem/progenitor cells to differentiate into various neuronal cell types. Such induced neurogenesis can lead to the treatment of a number of neurodegenerative disorders. Organically modiϐied silica (ORMOSIL) nanoparticles have shown a lot of promise for the delivery of genetic materials into the brain with high efϐiciency, without any sign of toxicity. In this chapter, I have discussed the effects of gene delivery using these nanoparticles in the brain. In addition, I have suggested various potential improvements in this technology that will drive its translation to the clinic. I have concluded with a brief discussion on the potential of these nanoparticles for the brain-speciϐic delivery of other therapeutic molecules, such as short interfering RNA (siRNA) and small molecule drugs.
