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Stem Cell System Shows How Gene Silencing Causes Fragile X Syndrome

 
• By Mark Ratner

Using stem cells, researchers have shown that the gene mutation that causes fragile X leads to gene silencing through the binding of a small repeat mRNA transcript to the gene itself. They further show that a small molecule can disrupt this RNA-DNA duplex, preventing the disease from occurring and suggesting a potential prenatal therapy.

Finally armed with the tools to investigate the onset of fragile X syndrome, the most common genetic form of intellectual disability, researchers have elucidated the intracacies of its mechanism and a potential new route for therapeutic intervention. Their work may also shed light on other genetic diseases similarly characterized by the silencing of a critical developmental gene.

A major difficulty in unraveling the mechanism underlying the onset of fragile X syndrome has been the lack of a...

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