The majority of the human genome encodes non-coding RNAs (ncRNAs), species of RNA without protein-coding potential but with powerful regulatory functions in disease onset and progression. Functional studies demonstrate that both coding and ncRNAs underlie various mechanisms in heart disease and that molecules targeting RNA species show promising efficacy in preclinical development. Accompanying the exciting developments in basic RNA biology, an equally provocative field has flourished for the design of RNA-based strategies to generate innovative types of therapeutics against these new "druggable" targets, going beyond our current repertoire of small chemistry or biologics. Here, we review the (bio)chemical basis of RNA-based drug design, provide examples that show promise as translatable drug products in preclinical studies, give an insight in the current barriers that hamper straight-forward clinical translation and discuss future directions that may overcome these hurdles to expand the current pharmacotherapy for myocardial disorders.
Keywords: Heart disease; Oligonucleotide; RNA; RNA therapeutics; RNA-based drugs.
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