In Fragile X Syndrome—the leading genetic form of intellectual disability and autism—the effects of a single defective gene ripple through a series of chemical pathways, altering signals between brain cells. It's a complex condition, but new research from Rockefeller University finds that inhibiting a regulatory protein alters the intricate signaling chemistry that is responsible for many of the disease's symptoms in animal models. The work, published in Cell, offers insight into how redundant mechanisms control the amount of protein in a cell and provides a path to possible therapeutics for the autism spectrum disorders.
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