RNA interference and current clinical applications: Review
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RNA interference (RNAi), which is one of the technological breakthroughs of modern biology not only became an important tool for gene function analysis in eucaryotes, but also being used as a promising technology for therapeutic gene silencing studies. The importance of this mechanism, which was described in almost all eukaryotic organisms, is reflected by the fact that the 2006 Nobel prize for medicine was awarded for the discovery of RNA interference by Craig Mello and Andrew Fire. Both microRNAs and siRNAs use RNAi machinery in order to be processed and being functional resulting in gene silencing via degraded or translationally repressed mRNA and no protein synthesis. During the last 15 years, a greater understanding of RNAi mechanism, the intracellular actioning, modifications for stable siRNAs and nanocarrier systems for delivery, presents siRNA therapeutics as an attractive newclass of therapeutics, especially against undruggable targets for the treatment of cancer and other diseases. According to American National Health Institute, there are many ongoing clinical trials with RNAi drugs. Current clinical studies are focused on developing therapies for the treatment of cancer, infection diseases, ocular conditions, cardiovascular and metabolic diseases, genetic diseases and a few other diseases. The drugs for the disease genes are designed in a way to reach to spesific tissue with high yield using carrier systems in order to achieve optimal efficiency. Thus, RNAi based drugs are expected to be an effective therapy system in near future, with ongoing active clinical applications. In this review, some of the ongoing clinical phase studies with siRNA therapeutics are summarized. © 2016 by Türkiye Klinikleri.