As a rapidly growing class of drug modalities, oligonucleotides are small (15–30 bp) nucleotide-based therapeutics which are capable of targeting DNA and RNA as well as other biomolecules. Oligonucleotides allow for precise and programmable targeting as they can theoretically be designed to selectively target any gene at RNA level.
RNA editing is a molecular process through which some cells can make discrete changes to specific nucleotide sequences within an RNA molecule after it has been generated by RNA polymerase. The technology offers tunable nucleotide change, limited off-site targeting and potential for undruggable targets.
mRNA is a long, polymeric molecule, composed of four different building blocks called nucleotides that translate genetic information from DNA to express proteins in cells to produce biological effects.