Small interfering RNAs: heralding a new era in gene therapy

Abstract

Last decades have witnessed a tremendous expansion in knowledge and availability of the genome sequence, which was of great importance for advancements in the field of gene therapy. This led to improved strategies based on use of nucleic acids with sequences complementary to specific target genes in treatment of many diseases. Especially, advancements have been achieved in discovery and use of diverse RNA molecules other than messenger RNAs (mRNAs), transfer RNAs (tRNAs), or ribosomal RNAs (rRNAs). Such RNA molecules, known as non-coding RNAs (ncRNAs), serve diverse biological roles some of which are still elusive. Some biological processes known to be regulated by ncRNAs include transcriptional regulation of genes, gene silencing, messenger RNA stability and translation, development, proliferation, haematopoiesis, apoptosis, protein translocation and chromosome replication. Up to now, the use of ncRNAs as a research tool has greatly improved gene therapy approaches for various diseases (Gallaso 2010), but also substantially improved drug discovery and target validation. In this book chapter, a focus on the use of a particular approach, namely RNAi for improved gene silencing for both, therapeutic approaches and identification of new therapeutic targets is discussed.