Discenza, Maria Teresa, and Jerry Pelletier. Insights into the physiological role of WT1 from studies of genetically modified mice. Physiol Genomics 16: 287-300, 2004; 10.1152/physiolgenomics.00164.2003.—The identification of WT1 gene mutations in children with WAGR and Denys-Drash syndromes pointed toward a role for WT1 in genitourinary system development. Biochemical analysis of the different WT1 protein isoforms showed that WT1 is a transcription factor and also has the ability to bind RNA. Analysis of WT1 complexes identified several target genes and protein partners capable of interacting with WT1. Some of these studies placed WT1, its downstream targets, and protein partners in a transcriptional regulatory network that controls urogenital system development. We review herein studies on WT1 knockout and transgenic models that have been instrumental in defining a physiological role for WT1 in normal and abnormal urogenital development.
Genetically modified mice for the study of apolipoprotein B