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Nat Biotechnol. 2017 Mar;35(3):222-229. doi: 10.1038/nbt.3802. Epub 2017 Feb 27.

Overcoming cellular barriers for RNA therapeutics.

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Department of Cellular and Molecular Medicine, University of California, San Diego, School of Medicine, La Jolla, California, USA.


RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs), antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR-Cas9, have great potential to target a large part of the currently undruggable genes and gene products and to generate entirely new therapeutic paradigms in disease, ranging from cancer to pandemic influenza to Alzheimer's disease. However, for these RNA modalities to reach their full potential, they first need to overcome a billion years of evolutionary defenses that have kept RNAs on the outside of cells from invading the inside of cells. Overcoming the lipid bilayer to deliver RNA into cells has remained the major problem to solve for widespread development of RNA therapeutics, but recent chemistry advances have begun to penetrate this evolutionary armor.

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