RNA is an attractive drug target with tremendous potential for future therapies of systemic and cancer-related pathologies. Only 1.5% of the human genome code for proteins yet more than 75% is transcribed into RNA. Thus, a large proportion of RNA is non-protein-coding including 3’UTRs of mRNAs with important regulatory functions. This makes them attractive drug targets considering that current therapeutics target only 700 proteins leaving most of the genome untouched. Especially cancer-related genes are often considered undruggable due to a lack of a classical drug binding pockets. Therefore, targeting these proteins via their mRNA 3’UTR region is an attractive alternative. We will establish a screening pipeline for small molecules targeting 3’UTR segments of cancer-related genes using binding and functional assays as well as RNA-based mRNA pulldowns. Integrative structural biology will reveal the structural basis for RNA binding activity and function of these compounds. Our acquired knowledge will pave the path towards design of molecular probes and future medicines to fight cancer.

Cíle udržitelného rozvoje

Masarykova univerzita se hlásí k cílům udržitelného rozvoje OSN, jejichž záměrem je do roku 2030 zlepšit podmínky a kvalitu života na naší planetě.