siRNA, a Powerful Tool for Gene Regulation
Small interfering RNA (siRNA) is a naturally existing mechanism through which cells regulate gene expression. By silencing specific genes siRNA can up/down regulate cellular pathways of interest. siRNA is a short double stranded RNA that mediates target messenger RNA (mRNA) breakdown and as a result alters protein expression/function. Therefore, siRNA has been widely used for research purposes and is currently a hotspot for clinical gene therapy. siRNA however, cannot be easily employed by scientists due to its high degradability and immunogenicity. Additionally, naked siRNA cannot efficiently transfect cells since it is not readily taken up by them due to unfavorable electrostatic interaction of charged siRNA molecules and hydrophobic cell membrane. Lipid nanoparticles (LNPs) provide a powerful tool for siRNA delivery. LNPs take advantage of protective lipid layers to shield the siRNA cargo, minimize toxicity and enhance endocytosis without any need for transfection reagents. LNP encapsulated siRNA can be used for screens, functional studies and in vivo therapeutic targeting in animal models. siRNA LNPs produced through Precision NanoSystems microfluidic mixing method have been proven highly efficacious in transfecting different cell lines and in vitro and in vivo gene knock-down.