Oligonucleotide therapeutics for central nervous system disease

Yutaro Asami, Takanori Yokota

Department of Neurology and Neurological Science, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University （TMDU）

Oligonucleotide therapeutics is attracting attention as a new modality because it can target intracellular RNA, which has not been targeted by conventional small molecule or antibody drugs.　Oligonucleotide therapeutics is also an excellent target for neurological diseases, and is currently under active development and clinical trials for a number of neurological diseases.　In this review, we introduce antisense oligonucleotides and siRNAs as the basic structures, and also describe a novel heteroduplex oligonucleotide（HDO） developed in our laboratory.　We have found that systemically administered HDO reaches the brain and efficiently regulates the target RNA in the brain, which will contribute to the future development of oligonucleotide therapeutics.

Address correspondence to Dr. Takanori Yokota, Department of Neurology and Neurological Science, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University（TMDU）（1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8510, Japan）