Gene silencing—down-regulating the expression of a specific gene-product, or protein—is an invaluable tool used by researchers to validate drug targets. Of the many ways scientists have attempted to silence genes, only one—the homologous mouse model, in which individual genes are knocked-out one by one from an animal's genetic code—unambiguously achieves complete and accurate silencing of genes, claims [Gene Expression Technologies Ltd.] (GET) CEO Cameron Macdonald, PhD.
But the mouse model is inflexible, requires months to develop, and cannot be extrapolated for use in humans. More malleable technologies for gene silencing-based drug target validation, such as RNA-based...