Research provides path to possible treatment for Fragile X Syndrome and other types of autism
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.
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.
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- 3rd November 2017
- News Medical