Scientists find a new way to reverse symptoms of Fragile X

Wednesday, May 20, 2020 - 13:10 in Health & Medicine

MIT scientists have identified a potential new strategy for treating Fragile X syndrome, a disorder that is the leading heritable cause of intellectual disability and autism. In a study of mice, the researchers showed that inhibiting an enzyme called GSK3 alpha reversed many of the behavioral and cellular features of Fragile X. The small-molecule compound has been licensed for further development and possible human clinical trials. From the mouse studies, there are signs that this compound may not have the same limitations of another class of Fragile X drugs that failed in human clinical trials a few years ago, says Mark Bear, the Picower Professor of Neuroscience, a member of MIT’s Picower Institute for Learning and Memory, and one of the senior authors of the study. GSK3 inhibitors might also be useful against other diseases in which GSK3 plays a role, including Alzheimer’s disease, he says. Florence Wagner, director of medicinal chemistry at the...

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