From small molecules to oligonucleotides

The targeting of RNA of therapeutic interest can be done in two ways:

  • an approach based on the use of oligonucleotides that recognize a sequence of the RNA targeted in a specific way and therefore presenting a very high level of efficiency and specificity.
  • an approach based on the use of ligands capable of interfering with the structure and/or function of the RNAs. Examples of structurally specific ligands (e.g., G-quadruplex RNA, bacterial riboswitch) have been identified and studied.

The two approaches are therefore complementary and can converge for an effective targeting strategy applicable to a large number of targets.