Manipulating nucleic acids - regulation of gene expression through higher order structure and chemical modification

Abstract

The genetic information stored in dsDNA is transcribed into mRNA, which is then translated into protein to express its function. Nucleic acid therapeutics, as a modality that acts directly on nucleic acids, is being developed for the treatment of intractable diseases. In recent years, various mechanisms that control the spatio-temporal expression of genetic information, such as non-protein-codding ncRNAs, higher-order structures of nucleic acids, and small chemical modifications, have been elucidate, and the development of artificial functional molecules targeting these mechanisms is underway. This paper reviews the topic of nucleic acid therapeutics and artificial functional molecules that artificially manipulate gene functions.