Medical Research Council - London Institute of Medical Sciences

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Remodelling a Solution
05 December 2017

Remodelling a Solution

Fragile X syndrome is a complex genetic disorder, causing a suite of developmental problems and intellectual disabilities. Defects in a single gene, known as FMRP, are linked to the disease, yet how they can trigger such a range of symptoms has been difficult to grasp. FMRP regulates many proteins involved in signal transmission between neurons, but drugs targeting this function seem ineffective at treating Fragile X. Researchers have now realised that FMRP is also associated with another class of proteins: chromatin remodellers, which modify DNA to control the regions that are activated. The mouse brains pictured show the increased levels of one such protein, Brd4 (in green) in individuals with Fragile X syndrome (lower panel) compared to healthy mice (upper panel). Blocking the action of Brd4 in mice alleviated the symptoms of Fragile X, suggesting that chromatin remodellers could be a promising target for treating this disorder.

Written by Emmanuelle Briolat

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