Oligonucleotide therapies use short, synthetic DNA or RNA sequences to bind to specific disease-causing RNA, directly modifying gene expression at the source. Rather than targeting proteins later, they prevent harmful proteins from being made or restore missing ones, making them highly specific, targeted treatments for genetic and rare diseases.

This novel technology can tackle genetic regions that traditional small molecule drugs cannot reach. Oligonucleotide technology has broadened the spectrum of possible therapeutic targets. By controlling protein expression via RNA interference, previously “undruggable” proteins that were unaffected by conventional small molecules can now be affected.  On the other hand it is a challenge for synthetic oligonucleotides to enter cells and they are easily degraded by the body if administered without chemical modifications or specific delivery vehicles (like nanoparticles or lipid carriers). 

In this symposium an overview will be given of the potential of oligonucleotide therapy for the treatment of a range of different diseases, on the challenges for delivery at the site of action  and also novel applications in cellular therapy will be shown.