Mesoporous Silica Nanoparticles Facilitate Delivery of siRNA to Shutdown Signaling Pathways in Mammalian Cells

Genetic diseases are illnesses caused by abnormalities in genes or chromosomes, including hemophilia, Huntington's disease, and cancer. They can be caused by both genetic and environmental predispositions. Small interfering RNA (siRNA) is a powerful tool to inhibit gene function because it can be easily applied to any therapeutic target, providing revolutionary potency and selectivity of improved targeted therapeutics.[1] However, poor intracellular uptake, instability, and non-specific immune stimulation are obstacles associated with current methods of siRNA oligonucleotide delivery. Therefore, developing a stable, highly effective siRNA delivery system with optimal cellular integration and low toxicity is of utmost importance. In the present study, we successfully use a nanocomplex assembly of polyethylenimine (PEI) and mesoporous silica nanoparticles (MSNs) to protect and deliver siRNA into human cells, and effectively shutdown both exogenous and endogenous gene signals. © 2010 Wiley-VCH Verlag GmbH & Co. KGaA.