Emerging Clinical Applications and Pharmacology of RNA
Part Part D. Medical Applications
Published Online: 15 MAY 2012
Copyright © 2012 Wiley-VCH Verlag GmbH & Co. KGaA
Encyclopedia of Molecular Cell Biology and Molecular Medicine
How to Cite
Barik, S. and Bitko, V. 2012. Emerging Clinical Applications and Pharmacology of RNA. Encyclopedia of Molecular Cell Biology and Molecular Medicine.
- Published Online: 15 MAY 2012
In all living cells, the RNA acts as an important intermediate of genetic information transfer between DNA and protein. RNA is generally single-stranded, and a flexible and versatile biopolymer that incorporates many pharmacologically desirable traits of DNA and protein without some of their disadvantages. It is water-soluble and nontoxic, can be produced by chemical synthesis as well as recombinant cloning, faithfully replicates the parental DNA sequence, can be converted to DNA or protein, possesses enzymatic self-cleaving activity, and makes specific RNA–RNA, RNA–DNA, and RNA–protein interactions. The biologically remarkable forms and functions of RNA, such as antisense RNA, ribozymes, RNA decoys, aptamers, small RNA, and their role in RNA interference and epigenetic chromosomal regulation, are all rooted in this fundamentally unique combination of biochemical and molecular traits. It is only recently that some of these properties of RNA have been exploited to interfere with or repair dysfunctional or harmful nucleic acids or proteins, and to induce therapeutic gene products in a variety of pathological syndromes or infectious diseases. First, second, and third generations of RNA drugs have produced promising results, raising new hopes in bringing RNA therapeutics from the bench to the bedside. In this chapter, the basic features and discoveries of RNA that are relevant to its clinical usage are described, and details provided of some emerging clinical applications of RNA.
- CCR, CXCR;
- IFN (Interferon);
- LNA (Locked Nucleic Acid);
- PNA (Peptide Nucleic Acid);
- RNA drug;
- RNA interference (RNAi);