Occurring with a frequency of 1 in 10,000 live births, Wilms' tumor is one of the most common solid tumors of children. The genetic basis of this tumor is highly complex and several loci have been shown to be associated with tumor formation. Thus far, however, WT1 is the only gene that has been isolated and proven to carry mutations within Wilms' tumors. During the last few years, a wealth of experiments has been carried out to address the function of WT1 as a tumor suppressor and developmental regulator. This review focuses on studies addressing WT1 function; new approaches to understand WT1 function in vivo and present transgenic data in which WT1 was driven ectopically using a CMV promoter are discussed. Our results suggest that ubiquitous expression of WT1 is not compatible with embryonic development.