教育講演
核酸医薬の基礎と臨床 総論・化学
キーワード:
oligonucleotide therapeutics,
antisense oligonucleotide,
siRNA,
chemical modification,
drug delivery system
2020 年 37 巻 3 号 p. 238-241
詳細
2020 年 37 巻 3 号 p. 238-241
Oligonucleotide therapeutics are based on chemically synthesized oligonucleotides which are in almost all cases chemically modified. They are classified into some categories by their action mechanisms, such as antisense oligonucleotides (ASOs) and RNA interference (RNAi) drugs. These oligonucleotides can bind to their target molecules such as mRNAs by Watson–Click base pairing and/or other interaction also including multipoint hydrogen bonding. By their definitive action mechanisms and their molecular size which are between small molecules and antibody drug, oligonucleotide therapeutics can be highly–specific drugs that can target molecules in cells. In this review, we outline the classification and action mechanisms of oligonucleotide therapeutics. In addition, we also introduce nucleic acid chemistry, which can make biologically–unstable nucleic acid molecules into effective therapeutic reagents.
