Molecular Medicine: The Role and Potential of RNAi Techniques

RNA interference is the sequence specific gene silencing technique which holds promises and a lot of potential to become the drug of choice for many unresolved diseases. During the past few years, important studies have been done in translating the potential of RNAi into therapies for human disease. Viral infections, cancer, neurodegradative diseases and dominantly inherited genetic disorders are top most among the list of those diseases, which have the greatest potential to be treated by RNAi techniques. The rational design of RNAi based drugs can improve the therapeutic properties based on the recent progress in understanding the mechanism of action. The introduction of chemical modifications into siRNA and the improved delivery strategies that associate with high specificity and efficiency are being discussed intensively.