HDO is composed as follows.
Antisense strand (DNA, Gapmer, PMO etc.): Has a medicinal effect
Carrier strand (RNA): Transport active strand (medicinal effect) to disease site
Ligand: By binding a substance that specifically binds to a specific receptor to the carrier strand, it transports the active strand to a targeted disease site.
- Gapmer is an antisense strand consisting of Wing region – Gap region – Wing region. Wing region contains modified nucleotide (MNT) to enhance enzyme resistance and binding ability to target RNA. Gap region contains at least 4 consecutive pieces of DNA that are recognized by RNase H.
- Mixmer is an antisense strand in which multiple types of nucleic acids are “mixed” and arranged. It is not recognized by RNase H and does not cleave the target RNA.