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 signalling chemistry that is responsible for many of the disease’s symptoms in animal models.

The research 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 work centres on a group of proteins–known as chromatin remodelling 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 leukaemia,” 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 remodelling 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.

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2018-03-22T14:32:40+00:00

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