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'Brutal test' shows partial protein can reverse Rett syndrome

By Anette Breindl
Senior Science Editor

Thursday, October 12, 2017

A Gordian knot-like approach may be feasible in gene therapy for Rett syndrome. Researchers at the British University of Edinburgh have demonstrated that treating mice with a "radically truncated" gene that encoded about a third of the full-length MeCP2 protein, which is mutated in Rett syndrome, allowed the animals to survive long term with minimal symptoms.

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