Perspectives on the future

In a time when biologists are facing the enormous challenge of decrypting genetic messages encoded by whole genomes, the RNAi technique represents a precious tool. The use of either synthetic or stably expressed siRNAs will greatly facilitate and accelerate genomewide functional screening in model organisms and navigation within the decrypted genetic landscape. Also, siRNA-mediated gene silencing will be one standard method in the studying of specific roles of proteins in integrated cellular pathways. However, although RNAi has turned out to be an efficient approach to downregulate viral activity in cultured mammalian cells, its application as a therapeutic approach in humans will only be feasible if all risks of unspecific responses and other side effects have been eliminated. This demands a better understanding of the RNAi-related processes, particularly the correlation between structural features of the natural miRNAs and their strictly specific mode of action. This, in turn, will provide a basic reference design of siRNAs to be used for therapeutic purposes. At last, an appropriate system of siRNA delivery in vivo still needs to be developed.

RNAi is now one of the largest growing fields in biology, pharmacology, medicine, and even chemistry. Although the mechanism by which dsRNA modulates gene expression is not at all clarified, the development of novel approaches to cure diseases related to gene expression has proceeded to the point at which in vivo experiments in animal models are afoot. The recent development of leukemia cells and other cancer cells resistant to the well-known small-molecule inhibitors is calling for novel treatments. Even though a cell might be able to evolve mutated proteins that evade a small-molecule protein inhibitors, this does not happen if the encoding mRNA is degraded. RNAi might have held many promises, as yet, there is no experience in patients. Without doubt, the time to celebrate significant achievements in the clinical trials will be forthcoming soon.

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