|
The remarkable ability of short sequences of synthetic RNA to interfere with messenger RNA and thereby silence the activity of specific genes has proved incredibly helpful to geneticists wrestling with the genetic functions. And the push to harness this RNAi for therapeutic use is now beginning to make headway [12]. Reverse genetics is the most effective way to assess the function of a gene, but so far there has been no general method for renverse gentics other than gene targeting [3], which is slow and costly. Antisense approaches, such as antisense oligonucleotide and ribozyme technologies, have also been useful in reverse genetics, but only a limited degree. By contrast, the promise of small interfering RNA (siRNA) technology to 'knock down' the expression of any gene in vertebrate cells is set to revolutionize reverse genetic approaches [11]. Even at this early stage of understanding the molecular mechanisms of RNAi technology for selective gene silencing, it is clear that RNAi will be a widely used tool for establishing the functions of the genes [14]. Improvements on the currently available protocols for RNAi are being made and the methods are being applied by thousands of investigators in diverse fields. With the advent of these methods has come an explosion of studies that have employed RNAi. The current status of RNAi as an experimental tool is such that many investigators are now aware of the technology, but most have not yet implemented it in their own studies. The development of RNAi kits by several companies will facilitate the implementation of RNAi methods by essentially any investigator, regardless of their knowledge of RNAi mechanisms [12]. As for uses of RNAi in medicine, its potential remains to be established. The application of gene therapy approaches for the treatment of specific diseases has progresses much more slowly tjan initially anticipated [57]. There are, of course, many potential gene targets for therapeutic intervention using RNAi [58]. Studies that employ RNAi to counteract a disease process in vivo are emerging. RNAi that targeted the FAS gene or the Hepatitis C virus genome protected mice from hepatitis. One might expect that the next demonstration of successful treatment of disease in mice will come from models of cancer and neurodegenerative disorders. Ongoing and future pre-clinical studies in animal models will hopefully help optimize RNAi therapeutics for diabetes in its infancy, early clinical studies are soon to begin assessing the use of this new class of therapeutics to tackle metabolic diseases and other diseases. Because of the potential of RNAi for therapeutic intervention, major efforts should be placed on preclinical studies using this technology [57]. Downloads | Glossary | References | Contact © 2012, Saie Mogre. All Rights Reserved. |
