核酸医薬総論

抄録

Remarkable advances have been made in chemical modification and drug–delivery systems that improve efficacy of therapeutics oligonucleotides therapeutics, such as antisense oligonucleotide (ASO) and small interfering RNA (siRNA). ASOs act through various silencing mechanisms including the degradation of mRNA (gapmer type ASO) and the modulation of alternative splicing patterns (splice switching oligonucleotide). Recent favorable outcomes in clinical trials for neurodegenerative diseases and muscular diseases such as spinal muscular atrophy and Duchenne muscular dystrophy indicate high clinical potency of oligonucleotide therapeutics.

Here we reviewed recent advances in basic properties and clinical applications of ASO and siRNA, and provide future perspective on oligonucleotide therapeutics.