Understanding early and cell-specific changes in Rett syndrome provides markers to monitor efficacy of interventions and entry points to understand the brain circuits driving the condition.
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This work lays the foundation and provides preclinical evidence for a therapeutic approach for Rett syndrome that increases MeCP2 and confers functional improvement.
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Characterizing the trajectory of molecular events leading to Rett syndrome opens new opportunities for novel therapeutic approaches.
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DBS results in a significant and long-lasting improvement to memory in Rett animal models strongly suggesting evaluating this approach to treat Rett patients.
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