Abstract

The majority of approved drugs target proteins, which are encoded by a very small fraction of the human genome. When a pathology is associated with so-called undruggable proteins, an alternative strategy should be sought. In the last twenty years, non-coding RNA molecules have been shown to perform a variety of crucial biological functions, including regulating gene expression, protecting chromosomes from foreign nucleic acids, and guiding telomere synthesis. In this context, targeting RNA with small molecules is emerging as a promising therapeutic approach in pathologies such as cancer, viral infections, and neurodegenerative disorders.

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