RNA based therapeutics

Abstract

Astounding progress has been made in the identification of genes responsible for cell growth, development, and neoplastic transformation. With this knowledge has come a natural desire to "translate" this information into new therapeutic strategies for many of the common maladies that afflict humankind. These include in particular cardiovascular, gastrointestinal, neurological, infectious, and neoplastic diseases. Traditionally, therapies for most of the diseases are chemical/herbal drugs. Recent advances in molecular biology have made nucleic acid-based therapies feasible and while still relatively unproven, they represent one of the most exciting new areas in biomedical research. Attempts at inserting genes into cells that either replace, or counter the effects of, disease-causing genes has been one of the primary ways in which scientists have tried to exploit this new knowledge. This technically complex, as yet largely unrealized endeavor is what most individuals think of when the terms "gene therapy" or "molecular medicine" are discussed. Nevertheless, alternative strategies for treating diseases at the gene level are being developed. The common goals of these various strategies, which are turning out to be as technically demanding as more traditional gene therapy, is to identify disease related genes and target them for "silencing." Because the numbers of maladies that might be treated by this approach are genuinely enormous, this is clearly a most important field of endeavor. In this review the main scientific and technical aspects of RNA interference are introduced and some of its potential clinical applications are discussed.