For this week’s episode of our “How Does It Work?” series, we dive further into exploring Oligonucleotide Therapies.
Therapeutic oligonucleotides are incredible precise drugs. They are single or double stranded DNA or RNA molecules made up of 10-50 smaller molecules called nucleotides. The sequence and 3D structures of these nucleotide chains determines how they interact with other molecules in the body.
For example, antisense oligonucleotides are a type of therapeutic oligonucleotide containing complementary sequences to those of specific mRNAs. These bind mRNA to block or modify the expression of disease-causing proteins. RNA interference therapies, on the other hand, use double-stranded RNAs to trigger the degradation of specific mRNAs.
Aptamer therapies work very differently. Instead, the oligonucleotide folds into a highly specific 3D structure that complements that of a biomolecule such as a disease-causing protein, to modify its activity.
Therapeutic oligonucleotides are chemically synthesized by growing nucleotide chains. This unlocks the remarkable potential to create individualized drugs or peptide conjugated forms that increase drug half-life and target specific tissues.
These developments provide hope that therapy for many rare or currently untreatable diseases will soon be possible through the use of precision medicine.
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