シンポジウム1：神経筋疾患の核酸医薬の最前線
核酸医薬の基礎と臨床 総論
キーワード:
oligonucleotide therapeutics,
antisense oligonucleotide,
exon skipping,
viltolarsen,
chemical modification
2021 年 38 巻 3 号 p. 257-260
詳細
2021 年 38 巻 3 号 p. 257-260
Oligonucleotide therapeutics belong to molecularly–targeted therapy as well as antibody drugs and gene therapy drugs do, and directly bind target molecule without gene expression. They can bind nuclear RNA and work in the cell, though the antibody drug targets are limited to cell surface molecules. Antisense oligonucleotides, small interfering RNA, decoy, aptamer, and CpG ologonucleotides are mojor subtypes of oligonucleotide therapeutics which are classifed by the mechnism of action. The improvement of chemical modifications such as backbone modificatins and sugar modifications have increased thier stability. As a result, many of FDA approved oligonucleotide therapeutics have been developed in past five years. In 2020, viltolarsen was approved by FDA as a first antisense oligonucleotide developed in Japan. In this abstract, we review the characteristics, classification, and chemical modifications of oligonucleotide therapeutics. Moreover, we outline their current status in clinical practice.
