mRNA, a New and Improved Tool for Gene Therapy
As an important part of gene therapy, gene delivery is a great topic of interest to researchers. In order to have a protein expressed, plasmids and viruses have been used in the past. However, virus/plasmid vectors have been associated with many complications and there is a great need for systems that deliver genes without them. Messenger RNA (mRNA) therefore, is the preferred form of protein expression gene therapy since it does not integrate into host genome and delivers the entire protein code in one sequence. mRNA can be in vitro transcribed and easily purified. It is however highly unstable and readily degraded in cell cultures and in vivo and therefore requires a delivery system to improve the transfection. Lipid nanoparticles (LNPs) are capable of encapsulating mRNA and inhibit its degradation as well as facilitate transfection and endocytosis. Precision NanoSystems has demonstrated that LNP encapsulated mRNA using the microfluidic technology can efficiently transfect many cell types such as astrocytes and lead to efficacious expression of protein of interest.