Using as a tool to identify Pharmacological Therapies for Fragile X Syndrome.

Drug Discov Today Technol

Department of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104 ; Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104.

Published: September 2012

Despite obvious differences such as the ability to fly, the fruit fly is similar to humans at many different levels of complexity. Studies of development, cell growth and division, metabolism, and even cognition, have borne out these similarities. For example, bearing mutations in the fly gene homologue of the known human disease Fragile X, are affected in fundamentally similar ways as affected humans. The ramification of this degree of similarity is that , as a model organism, is a rich resource for learning about human cells, development and even human cognition and behavior. has a short generation time of ten days, is cheap to propagate and maintain and has a vast array of genetic tools available to it; making an extremely attractive organism for the study of human disease. Here, we summarize research from our lab and others using to understand the human neurological disease, called Fragile X. We focus on the model of fragile X, its characterization, and use as a tool to identify potential drugs for the treatment of Fragile X. Several clinical trials are in progress now that were motivated by this research.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3667993PMC
http://dx.doi.org/10.1016/j.ddtec.2012.09.005DOI Listing

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