Oligonucleotide

An oligonucleotide, or oligo for short, is a short molecule composed of nucleotides, the basic units that make up DNA or RNA. The term oligonucleotide derives from the Greek word “olígoi,” meaning “few” or “small,” and nucleotides, which are the building blocks of DNA and RNA. 

Unlike most drugs that act on proteins, oligonucleotides aim to correct errors in the genetic code. Oligonucleotides can be synthetically fabricated in a lab, producing a short sequence of nucleotides that binds to DNA or RNA, like a key fitting a lock. This technology allows for the treatment of disorders that were previously untreatable and targets the genetic issue at the root of the disease.