Understanding and treating diseases with RNA
Ribonucleic acid (RNA) plays a central role for the understanding of diseases and finding treatments. Using Spinal Muscular Atrophy as an example, we show how lack of processing of an RNA in neurons triggers the disease, which is treated with a small therapeutic RNA.
Basic research is indispensable for the understanding of diseases and finding treatments. The central role that RNA plays is exemplified by Spinal Muscular Atrophy (SMA). SMA is the first neurodegenerative disorder for which a disease-modifying therapy was approved. Interestingly, both the drug – an antisense oligonucleotide, and its target – the so-called pre-mRNA of the defective SMN gene, consist of RNA.
Experience cutting-edge technology during a live Nanopore sequencing demonstration at the booth of the research network “Swiss Initiative for RNA & Disease”. Literally grasp the concept of genetics while handling building blocks to simulate molecular editing (splicing) and manually bring pre-mRNA into its final form. Test your scientific thinking skills by logically arranging research questions, experimental approaches, results and corresponding new hypotheses.
