RNAi: A CHALLENGING PROSPECT IN THERAPEUTICS

RNA interference (RNAi) represents a mechanism invented by nature to protect the genome. In the past few years the field has emerged at a surprisingly high pace. RNA interference (RNAi) has advanced from its basic discovery in lower organisms to becoming a powerful genetic tool and perhaps our single most promising bio therapeutic for a wide array of diseases. Numerous studies document RNAi efficacy in laboratory animals and the first clinical trials are underway and thus far suggest that RNAi is safe to use in humans. RNAi is a posttranscriptional process that can effect gene silencing through chromatin remodeling, blocking protein synthesis and cleaving specifically targeted mRNA. Since RNA interference has only been recently discovered, there are many possible future avenues for application. Its specificity makes it an ideal tool for knocking down single genes for studying gene function or for gene therapy. There appear to be numerous potential clinical and medical applications. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. Silencing of gene expression by siRNA is rapidly becoming a powerful tool for genetic analysis and represents a potential strategy for therapeutic product development. We highlight emerging solutions and concurrently discuss novel therapeutic RNAi-based concepts. The therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise. The current rapid advances create realistic optimism that the establishment of RNAi as a new and potent clinical modality in humans is near. Sheeja S Rajan * et al. /International Journal Of Pharmacy & Technology IJPT | Dec-2010 | Vol. 2 | Issue No.4 | 419-445 Page 420