While no animal model can replace a human, the availability of several varieties of genetically modified mice has been essential to researchers who are working to better understand Down syndrome. Mice, however, offer limited opportunities, especially in the area of behavioral testing and in depth neurological analysis.

Scientific research in Down syndrome is moving quickly, and researchers’ need for more sophisticated animal models is critical. The Jerome Lejeune Foundation has received matching funds from the Betancourt Foundation to develop the first rat model of Down syndrome. Work is underway to develop 7 models of genetically engineered rats that will mimic Down syndrome and provide researchers new tools to study 3 critical gene targets: APP, DRK1A, and CBS. This new rat will make possible new opportunities to study behavioral changes and cognitive status, reinforcing the understanding of these genetic pathways that are altered in Down syndrome.

A rat will also make more advanced testing possible to researchers than are currently possible with mice. Rats provide a remarkable advantage in the study of the physiology of the central nervous system, and for conducting neuropharmacological studies leading to drug treatments to improve the lives of those living with Down syndrome.

(Rat photo By AlexK100 [CC BY-SA 2.0 (http://creativecommons.org/licenses/by-sa/2.0)], via Wikimedia Commons)