Basic principles and clinical applications in oligonucleotide therapeutics

DOI
  • Matsubayashi Taiki
    Graduate School of Medical and Dental Sciences, Department of Neurology and Neurological Science
  • Yokota Takanori
    Graduate School of Medical and Dental Sciences, Department of Neurology and Neurological Science

Bibliographic Information

Other Title
  • 核酸医薬の基礎と臨床　総論

Description

<p>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.</p>

Journal

Keywords

Details 詳細情報について

  • CRID
    1390009305281274624
  • NII Article ID
    130008142609
  • DOI
    10.15082/jsnt.38.3_257
  • ISSN
    21897824
    09168443
  • Text Lang
    ja
  • Data Source
    • JaLC
    • CiNii Articles
  • Abstract License Flag
    Disallowed

Report a problem

Back to top