080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

New Drug Strategy Against Fragile X Syndrome Identified

by Kathy Jones |

A potential new strategy for treating fragile X syndrome, the most common inherited cause of intellectual disability has been identified by researchers at Emory University School of Medicine.

The researchers have found that a class of drugs called phosphoinositide-3 (PI3) kinase inhibitors can correct defects in the anatomy of neurons seen in a mouse model of fragile X syndrome. In experiments with cultured neurons from the hippocampus, a brain region involved in learning and memory, the drugs could restore normal appearance and levels of protein production at synapses, the junctions between cells where chemical communication occurs. The results, published online this week in the Journal of Neuroscience, suggest that PI3 kinase inhibitors could help improve learning and cognition in individuals with fragile X syndrome.

“This is an important first step toward having a new therapeutic strategy for fragile X syndrome that treats the underlying molecular defect, and it may be more broadly applicable to other forms of autism,” says senior author Gary Bassell, professor of cell biology and neurology at Emory University School of Medicine. He adds that his group has recently begun experiments in the mouse model to assess PI3 kinase inhibitors’ effects on behaviors associated with fragile X syndrome.

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