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Dis Model Mech. 2011 Sep;4(5):596-606. doi: 10.1242/dmm.007716. Epub 2011 Aug 4.

The use of mouse models to understand and improve cognitive deficits in Down syndrome.

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Department of Physiology and McKusick-Nathans, Institute for Medical Genetics, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.


Remarkable advances have been made in recent years towards therapeutics for cognitive impairment in individuals with Down syndrome (DS) by using mouse models. In this review, we briefly describe the phenotypes of mouse models that represent outcome targets for drug testing, the behavioral tests used to assess impairments in cognition and the known mechanisms of action of several drugs that are being used in preclinical studies or are likely to be tested in clinical trials. Overlaps in the distribution of targets and in the pathways that are affected by these diverse drugs in the trisomic brain suggest new avenues for DS research and drug development.

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