Health
The theoretical appeal of RNA drugs are tempered somewhat by the multiple barriers that impede their development. Now, a large scale international collaboration based at the University of Copenhagen is paving the way toward safe and effective drugs for the treatment of human disease
hitting the target RNA drugs:
The traditional approach to treating genetic and infectious diseases and disorders involves the targeting of relevant proteins. These proteins differ widely from one to the next, and the drugs (e.g. antibodies or small molecules) that target them are likewise unique; one cannot simply take the blueprint of one drug as the basis for another. Since the late 1970s, however, the potential of RNA drugs has provided a promising alternative that has kept scientists busy the world over. Heading straight for the messenger RNA (mRNA) that encode proteins,
RNA drug development benefits from the similarities that exist between these RNA molecules. The knowledge that enables the design of one RNA drug, therefore, is to some extent transferable. No longer does each therapeutic need to be entirely considered anew. Despite the great strides that have been made, the potential of RNA drugs is yet to be fully realised as major challenges exist concerning off-target effects, tolerability and the potency of these therapeutics. Today, these issues are at the forefront of the work being carried out at the Center for Computational and Applied Transcriptomics (COAT). A newly established cross-disciplinary enterprise, COAT aims to facilitate the design of safe and effective RNA drugs through the integration of experimental biology data and cutting edge computational methods.
“Although drugs are designed with specific mRNA targets in mind, there can be a
tendency to stray off target and include other genes in their effects”
Led by Anders Krogh, a professor in bioinformatics and head of the University of Copenhagen’s Section for Computational and RNA Biology, COAT brings together the expertise of the Danish universities of Copenhagen and Aarhus, the University of California Santa Cruz (UCSC), New York’s Memorial Sloane Kettering Cancer Center (MSKCC) and the biopharmaceutical company Roche Innovation Center Copenhagen (RICC). Enabled by a €5m grant from the Danish Strategic Research
Council, the centre’s work marks a vital contribution toward the functional annotation of the human genome, an endeavour that promises to narrow the gap in our understanding and treatment of genetic diseases in humans.
24 Insight Publishers | Projects
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