Advancing Gene Therapy
Gene therapy is a great focus of interest due to its high specificity and low anticipated off-target effects. Unlike small molecule drugs, gene therapy enables researchers to specifically target a protein of interest and/or its variants and therefore is highly customizable. Two desirable tools of gene therapy are small interference RNA (siRNA) and messenger RNA (mRNA). siRNA provides an invaluable means of specifically silencing genes and inhibiting protein expression, thus regulating downstream cellular pathways. mRNA on the other hand provides the full template for expression of protein of interest. Both systems are great assets to research and are potential clinical therapeutics for treatment of genetic disorders. Precision NanoSystem provides flexible tools to ensure optimal gene delivery for both siRNA and mRNA for in vitro and in vivo purposes. Through Precision NanoSystems’ products, researchers can use pre-designed kits or apply nucleic acids and formulation material of their own to achieve advanced gene targeting for silencing or expression of genes of interest.