Using Drosophila as a tool to identify pharmacological therapies for fragile X syndrome

Sean M.J. McBride, Sandra L. Holloway, Thomas A. Jongens

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


Despite obvious differences such as the ability to fly, the fruit fly Drosophila melanogaster 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, Drosophila 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 Drosophila, as a model organism, is a rich resource for learning about human cells, development and even human cognition and behavior. Drosophila 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 Drosophila an extremely attractive organism for the study of human disease. Here, we summarize research from our lab and others using Drosophila to understand the human neurological disease, called fragile X. We focus on the Drosophila 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.

Original languageEnglish (US)
Pages (from-to)e129-e136
JournalDrug Discovery Today: Technologies
Issue number1
StatePublished - Mar 2013
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Drug Discovery


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