New modalities that control human diseases at the RNA level

DOI
  • Inoue Takao
    Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences

Bibliographic Information

Other Title
  • “RNA”を制御するモダリティの開発動向

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Description

In recent years, the clinical development of oligonucleotide therapeutics, such antisense oligonucleotides and siRNAs, have been progressing rapidly, and they are attracting attention as new modalities for genetic diseases and intractable diseases for which there have been few therapeutic options. In addition, the development of the COVID-19 vaccine has brought the medical application of mRNA into the limelight, and it is being recognized as a next-generation modality. These therapeutics have in common that they control human diseases at the level of RNA, and the fact that they generally have high medicinal efficacy has made them an area of high interest. In this paper, we will review the overall picture of these modalities and summarize the development trends and characteristics of them.

Journal

  • MEDCHEM NEWS

    MEDCHEM NEWS 31 (4), 174-179, 2021

    The Pharmaceutical Society of Japan

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