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Archive - Nov 3, 2017

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Potential New Treatment for Fragile X Targets One Gene to Affect Many

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 the Septmber 7, 2017 issue of 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. The article is titled “Excess Translation of Epigenetic Regulators Contributes to Fragile X Syndrome and Is Alleviated by Brd4 Inhibition.” The work centers on a group of proteins--known as chromatin remodeling proteins--that control gene expression. Chromatin remodelers work by adding chemical tags to DNA, regulating the cellular machinery that transcribes genes into messages. "Drugs that target chromatin remodelers are already in clinical trials to treat cancers like leukemia," says study author Dr. Erica Korb, a postdoctoral researcher at Rockefeller. "It is an attractive approach because a single inhibitor allows you to target a whole network of genes at once." The new research suggests that chromatin remodeling proteins may similarly play a key role in Fragile X Syndrome. By targeting chromatin remodelers in animals, the scientists were able to successfully alleviate symptoms of the disease. Researchers have known for some time that Fragile X Syndrome is caused by defects in a single gene, known as FMRP, but exactly how FMRP affects neural function has remained a mystery. A break came in 2011, when Rockefeller's Dr. Robert B.