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RNA Editing Repairs Rett Syndrome Mutation in Mice

Rett syndrome is a lifelong, genetic neurological disorder characterized by severe impairments including regression of speech, the appearance of seizures, and respiratory abnormalities. A hallmark of the disease is constant, repetitive hand movements.

A new study shows that RNA editing may repair the underlying cause of Rett Syndrome in a mouse model. The technology recoded enough RNA to restore half of the normal protein in three different kinds of neurons in the Rett mouse, demonstrating that programmable RNA editing can be utilized to repair mutations in mouse models of neurological disease.

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