A decade and a half after being first described to occur in mammalian cells, numerous trials are indicating that RNA interference can be harnessed to treat human disease. This article argues that positive results are now starting to emerge for the application of vector-based ddRNAi.
Despite an investment of billions of US dollars in the search of novel therapies, cancer still remains the leading cause of death in the world. This emphasises the need to identify novel tumour dependencies and molecular targets.
RNAi screening is arguably the fastest growing field with the premise to better understand gene function at the genome level. It has been hailed as the second genomics wave, and in combination with the human genome-sequencing projects, would constitute the holy grail of modern genetics.
Libraries of siRNAs directed against predefined subsets of genes now offer the capacity to greatly accelerate and improve the quality of functional genomics based drug discovery by enabling a much more targeted approach that effectively integrates the discovery and validation of novel targets.
RNAi (RNA interference) is an important target validation tool that has risen to prominence since 2001. For a number of reasons, it has supplanted other pre-existing target validation tools and is now the method of choice for target validation in cell culture systems.