1 Future of RNAi in Medicine: A Review

Abstract: The discovery of 21-23 nucleotide RNA duplexes, called small interference RNA (siRNA) may well be one of the transforming events in biology in the past decade. RNAi can result in gene silencing or even in the expulsion of sequences from the genome. Efforts to understand its mode of action have revealed a central role in gene regulation and host defense. The specificity, efficiency and potency of RNAi make it an attractive tool for analyzing the function of genes. RNA interference can be exploited artificially to inhibit the expression of any gene of interest. RNA interference systems could be used clinically to suppress gene expression as a therapeutic strategy in many diseases characterized by elevated gene function. Finally, as a therapeutic tool, it has shown enormous promise in the control of a large array of diseases. This review focuses on the potential therapeutic use of RNAi for various diseases, the current understanding of RNAi biology and how RNAi has been utilized to study the role of different genes in the pathogenesis of cancer, HIV, infectious diseases, HBV, cardiovascular and cerebral diseases, neurogenerative diseases, malaria, etc