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Stem Cell System Shows How Gene Silencing Causes Fragile X Syndrome

This article was originally published in Start Up

Executive Summary

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.

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