Rna Interference: From basics to Therapeutics

RNA interference (RNAi) is a powerful experimental tool and having a good potential for development of therapeutics. RNAi is a sequence specific mechanism to control the expression of the target genes. This technique has proven its potentials both in-vivo and in-vitro. RNAi based strategies are having capabilities to move from bench to bedside. There is need of precise tools for designing the siRNAs in order to get the effective knock down of the target genes. However, the aspects of off-target effects, delivery methods, induction of immune response, and dose determination for delivery should be considered carefully. The main hurdle for using RNAi-based therapy is the effective delivery of RNAi based drugs to the target cells or tissues in vivo. If these challenges associated with siRNA can be met then potentials of RNAi could be exploited for the development of RNAi based therapeutic tools. The assessment of safety and efficacy related to the use of siRNA is a matter of paramount importance.