Current trends of development of oligonucleotide therapeutics

Takao Inoue

Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences

Oligonucleotide therapeutics, such as antisense oligonucleotide and siRNA, are different from conventional small molecule drugs and antibody drugs in that they target RNAs not proteins.　The development of oligonucleotide therapeutics for intractable and hereditary diseases has been the main focus, but in recent years, clinical development of oligonucleotide therapeutics for conditions with a large number of target patients, such as lipid metabolism disorders and cancer, has also progressed.　In this review, the definition, classification, basic properties, and mechanism of action of oligonucleotide therapeutics are explained, and examples of their practical application are introduced.

Address correspondence to Dr. Takao Inoue, Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences（3-25-26 Tonomachi, Kawasaki-ku, Kawasaki, Kanagawa 210-9501, Japan）