2020 Volume 37 Issue 3 Pages 242-244
Recent progress in biotechnology has been accelerating the development of new modality drugs, including monoclonal antibodies, peptide drugs and cell–based therapeutics. Nucleic acid therapeutics, the subject of this manuscript, are negatively charged macromolecules consisting of tens or more nucleotides. This physicochemical property greatly determines their interaction with biological components, such as circulating cells in the blood and cells in organs, and serum proteins, which then determines their tissue distribution. To increase the binding affinity to target molecules, such as mRNA, as well as to increase the biological stability, most nucleic acid therapeutics consist of various types of nucleic acid derivatives or analogues. Then, they will exhibit unique tissue distribution profiles depending on their physicochemical properties. Drug delivery systems, which are intended to increase the potency of pharmaceuticals, have been developed also for nucleic acid therapeutics. They include : nanoparticles, galactose modification, and nanostructuration. Success in the control of the tissue distribution of nucleic acid therapeutics will further increase their therapeutic usefulness for various diseases, including neurological diseases.
