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RNA as a drug target offers a wealth of opportunities to therapeutically modulate numerous cellular processes, including those linked to the so-called “undruggable” protein targets. Of particular interest is the modulation of the natural process of pre-mRNA splicing, to control the formation of the corresponding protein products. A successful example of this approach, which will be reviewed here, is the modulation of the SMN2 alternative splicing for the treatment of spinal muscular atrophy (SMA).

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