Current State of Oligonucleotide Therapeutics
Oligonucleotides are a relatively new class of drugs, composed of natural and synthetic nucleotides, which primarily include small interfering RNA (siRNA), micro RNA (miRNA), and antisense oligonucleotide (ASO). These molecules achieve therapeutic effects through RNA interference, degradation, or splice-modulating pathways.1, 2, 3, 4 Other oligonucleotide therapies include messenger RNA (mRNA, single strand, >500 mers),5 small activation RNA (double strand, ~20 mers),6 antagomir (single strand, ~20 mers),7 and aptamer (single strand, >30 mers).8 This article focuses on synthetic ASOs (single strand, 16–22 mers) and siRNAs (double strands formed by hybridization of a pair of complementary sense and antisense strands, 19–25 mers).
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