RNA therapeutics at a glance
For many years the focus of therapeutic developments and scientific research was directed at the genome and DNA-related proteins. Yet, there is a completely different level of cell regulation – transcriptome level, presented by RNAs. RNA molecules are not just intermediates between DNA and proteins, in fact, they carry enzymatic functions, control gene expression, and build up proteins from amino acids.
Major breakthroughs in transcriptomics have been made since the beginning of the century: the discovery of RNA interference in 2001 blew up the industry and was awarded the Nobel Prize in 2006. RNA interference is a gene silencing mechanism promoted by small siRNA molecules that are also exploited in patisiran and inotersen – first-in-class RNA therapeutics. Both drugs target transthyretin, a protein involved in the pathogenesis of transthyretin-mediated amyloidosis, and reduce the expression of its mutant version.
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