Page 2 - Oligonucleotide Therapeutics Solution Guide

Types and Characteristics of Oligonucleotide Therapeutics

Oligonucleotide therapeutics are nucleic acid polymers generally comprised of a few to several dozen bases However, it is a practical problem that oligonucleotide therapeutics are degraded and excreted rapidly after
(including modi ed bases) linked together. They are produced by chemical synthesis and act directly on organisms administration by exonucleases and endonucleases that are abundant in blood and cells. This problem is being
without being translated into proteins. Oligonucleotide therapeutics are characterized by the ability to target addressed by the introduction of modi ed oligonucleotides to improve chemical stability in vivo and by the
speci c diseases. Another advantage is that it takes less time than conventional methods to nd new therapeutic development of lesion-targeted DDS (drug delivery systems) technologies.
candidates because oligonucleotides are easy to design and synthesize.

Inhibit functionality by binding to a target protein Inhibit gene expression by binding to target RNA

Aptamer Decoy Antisense Ribozyme siRNA
This is a single strand of DNA or RNA This binds to proteins called
that forms a thermodynamically stable transcription factors that regulate This inhibits gene expression by This is an RNA with enzymatic This is a double-stranded RNA of
conformation through complementary gene expression, preventing binding to the sense chain mRNA activity that is coordinated by about 21 ~ 23 bases. It inhibits
sequences within the molecule. them from binding to the genome encoding the target protein. metal ions. It inhibits protein protein expression by degrading
It binds directly to and acts on and signaling an immune expression by binding to and mRNA of target genes.
target proteins. response. cleaving target mRNA.
Target Protein

Inhibition of signal Inhibition of DNA transcription
transduction and Gene Expression miRNA

RISC
Complex
Transcription
factors
DNA mRNA

Protein

TLR9
Endosome
miRNA
Nucleus

Cell

CpG oligo
miRNA
This is single-stranded DNA with a sequence Show ef cacy by activating Silence target gene expression by
of cytosine and guanine linked by miRNAs are regulatory RNAs that
phosphodiester bonds (CpG motif). It is taken innate immunity regulate gene expression. supplying miRNA
up by endocytosis and activates innate Silence target gene expression
by supplying miRNA.
immunity by acting on Toll-like receptors
9 (TLR9) expressed on
endosomal membranes. Note: Not all oligonucleotides therapeutics are applicable.