RNA as Target & Drug

RNA biology has been one of the fastest developing areas in the life sciences over the last decades. Concomitantly, increasing numbers of alterations in RNA metabolism that contribute to disease pathology are being discovered. These include for example genetic diseases, cancer, as well as metabolic and neurodegenerative diseases. The NCCR RNA & Disease aims at unraveling the role of RNA in disease mechanisms. It is therefore seeking collaborations with clinicians and also offers to consult clinicians about questions relating to RNA research. In addition to disease-associated alterations in RNA metabolism, which represent promising drug targets, RNA can also be used as a therapeutic in the form of highly specific antisense oligonucleotide drugs.  In 2016, the FDA approved two oligonucleotide drugs designed to change the alternative splicing pattern of their respective mRNA targets: one for the treatment of Spinal Muscular Atrophy (SMA) and the other for the treatment of Duchene Muscular Dystrophy (DMD). Clinical trials with additional oligonucleotide drugs are currently ongoing.

If you want to explore the possibility of collaboration or have a question related to RNA research, please write an email to officewhatever@nccr-rna-and-disease.ch, and your email will be forwarded to the principal investigator of the NCCR RNA & Disease network who is best suited to assist you.