Target validation using human tissue: from gene expression to function

Arguably, one of the main reasons for the pharmaceutical industry's current poor performance in bringing successful new drugs to market is its over-reliance on animal data in the discovery and development processes. We propose that more use should be made of donated human tissues in order to identify and validate drug targets and to discover and develop novel drugs to act upon them. We review the ways that human tissues may be used for this purpose, from the measurement of gene expression, through determination of specific protein abundance, to functional evaluation, both at the level of tissue and specific cell type. The proposal is not simply theoretical, as we provide a specific case history to illustrate these points.