RNAi technique, how far is it from pediatrics?

Abstract

The new technology of ribonucleic acid interference (RNAi) or small/short interfering RNA (siRNA) can be used to reduce expression of genes in a sequence specific manner, and thereby can treat various diseases caused by expression or overexpression of genes. Phase 1 and phase 2 clinical studies on application of this technology to treat diseases have demonstrated efficacy and safety of this approach in a few specialties/subspecialties. However, no clinical trials have been reported in the fields of pediatrics. This article aimed to describe very briefly what the RNAi technique is, examples of demonstration of the efficacy and safety of RNAi techniques in a few different fields of clinical medicine, and to encourage pediatricians and pediatric researchers to actively participate in studies on this new therapeutic approach for treatment of various pediatric diseases.