Stephen T. Warren

Discovered triplet repeat expansion as a new heritable mechanism of disease and the cause of fragile X syndrome, the most common inherited form of cognitive deficiency. He showed that fragile X mental retardation protein (FMRP) is an mRNA-binding protein that associates with polyribosomes to regulate the translation of specific mRNAs. He performed a high-throughput chemical screen in Fmr1 knockout flies and discovered new therapeutic targets, leading to an ongoing, multicenter phase II clinical trial in patients with Fragile X Syndrome.