New approaches for the development of nucleic acid-based therapeutics: Challenges and opportunities in RNA targeting

This article presents a brief overview of both existing and new, emerging strategies for RNA-mediated gene silencing that can potentially be applied as highly selective approaches against abnormal gene expression. Despite the availability of numerous approaches for controlled translational arrest of disease-relevant proteins, none has gained superiority thus far. Therefore, the purpose of this review is to highlight some new strategic directions, which may inspire development of novel therapeutic approaches and thus allow expanding the therapeutic window for drug discovery. Our particular interest is focused on peptidyl-oligonucleotide chemical ribonucleases, although some other approaches for sequence-specific RNA targeting are also presented.