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Diseases & Treatments

Alternative gene-therapy approaches take aim at Rett syndrome

spectrumnews.org
 2021-06-16

Cover picture for the articleIn theory, researchers know how to reverse many of the debilitating effects of Rett syndrome: Boost expression of the protein MECP2. People with the condition typically have a mutated copy of the MECP2 gene and, as a result, deficient levels of its protein. This loss leads to intellectual disability, autism, seizures and trouble walking, talking and breathing.

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