Functional genomics and target gene validation in experimental and human disease

Drug Discov Today Technol. 2004 Oct;1(2):105-11. doi: 10.1016/j.ddtec.2004.08.007.

Abstract

In contrast to rare monogenic disorders related to mutations in distinct genes, frequent multifactorial diseases including complex neuronal disorders as well as neoplasms involve expression changes in a multitude of genes. The integration of data derived from human disease tissue and of animal models by data mining constitutes a recent approach to increase the pathogenetic significance of target genes. Functional validation by animal models represents a key step to develop new treatment pathways for common disorders.: