Working in cell cultures, researchers show how experimental drug blocks genetic trigger behind fragile X syndrome
Researchers report that they’ve found the gene-silencing mechanism that causes fragile X syndrome, the most common wholly genetic cause of autism. They also describe reversing the problem in cell cultures using an experimental drug. The findings offer early promise that a similar approach might prevent or treat the root cause of fragile X syndrome in people.
“This is cutting-edge science that uncovers new details about the precise cause of fragile X and is new territory in the search for treatments aimed at its genetic cause rather than the symptoms,” comments Daniel Smith, Autism Speaks senior director for discovery neuroscience. Dr. Smith was not involved in the research. “It will be essential to determine whether these findings in a cellular model reflect the actual human condition and have the potential for truly new treatments,” he says….