On November 18, 2002 Cancer Research Technology Limited ("CRT") and the University of Cambridge (UK) reported a collaboration with Cyclacel Limited and a top 5 Pharmaceutical company to develop tools for target validation and drug discovery based on the technique of RNA interference (Press release, Cancer Research Technology, NOV 18, 2002, View Source [SID1234523466]).
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The term RNA interference ("RNAi") describes the cellular response to double-stranded RNA that results in sequence-driven gene specific silencing. Originally established as an important tool for functional genomics in the nematode Caenorhabditis elegans and the fruitfly Drosophila melanogaster, the potential for this technology was recently recognised in higher eukaryotes. In late 1999, Dr. Magdalena Zernicka-Goetz and colleagues (Dr. Florence Wianny, Professors David Glover and Martin Evans) at the University of Cambridge (UK) provided the first definitive demonstration of RNAi-mediated inhibition of endogenous genes in mammalian cells. This work in oocytes and early stage embryos sparked a flurry of research in the area, culminating recently in the use of short interfering double stranded RNA (siRNA) to achieve knockdown of a wide range of mammalian genes, and the development of specific vectors to express these effectors.
There are a number of published patents on techniques for performing RNAi, and undoubtedly more applications in the pipeline. CRT is the holder of two key patent applications in the field, one of which relates to the work of Dr. Zernicka-Goetz and colleagues. The second patent filing from CRT covers pSUPER, a vector for long-term expression of RNAi effectors in mammalian cells, developed at the Netherlands Cancer Institute (Amsterdam) in the group of Professor Rene Bernards. CRT is offering licenses to this portfolio of intellectual property, branded the SUPER RNAiTM System, for target validation, drug discovery and therapeutics. Several pharmaceutical and biotechnology companies are working with CRT towards generating RNAi-based systems to meet the current demands of the industry for higher throughput analysis of mammalian gene function and rapid production of disease models to aid drug discovery. In addition, CRT is exploring the potential for a new class of therapeutics, using RNAi for the inhibition of genes that are aberrantly expressed in cancers.
The collaboration announced today is funded in part by Cancer Research UK. The partners aim to build on the pioneering work in the Cambridge laboratory, and the recent exciting discoveries in the field, to establish RNAi as a viable tool for transient or persistent gene knockdown in mammalian cells and whole animals (e.g. the mouse). The successful development and optimisation of RNAi in mammalian systems will facilitate gene analysis in the academic laboratory, meanwhile use of this technology for commercial target validation and drug development will accelerate the gene-to-drug process.