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RNA World : a new frontier in biomedical research

Executive Summary

Recent years have witnessed an unprecedented gain of knowledge from ribonucleic acid (RNA) research.

This caused a paradigm shift from the 40-year-old central dogma that RNA merely serves as a messenger in genetic information transfer to a view that RNA not only plays a role in a multitude of cellular functions, for example in the regulation of gene expression, but also could be the key molecule that led to the origins of life on Earth – the so-called RNA World hypothesis. High expectations and great hype arose from the discovery of RNA interference (RNAi; a mechanism widely employed by eukaryotic cells to inhibit protein production at a
post-transcriptional level) which allows gene silencing in experimental settings and has enormous therapeutic potential. RNAi established itself very quickly as a useful molecular biology tool making large-scale functional genomics screens and high-throughput drug target screening possible. There is increasing optimism that RNA-based approaches will bring significant advances to the diagnosis, treatment and prevention of a multitude of human diseases. The exploitation of RNA molecules as diagnostic tools and as therapeutic agents has only begun.

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