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Significance Rett syndrome (RTT) is an X-linked neurodevelopmental disorder caused by mutations inMECP2 . To treat RTT, reactivating the dormant copy ofMECP2 on the inactive X has been of considerable interest. Although potential therapeutics have been identified, their development has been hampered by the lack of a suitable female mouse model and uncertainty regarding how much MECP2 needs to be restored. Here, we create a female model with a more severe phenotype, including a short lifespan, neuromotor impairment, and repetitive behaviors often seen in human RTT. Significantly, 5–10% MECP2 restoration in brain extends lifespan by eightfold and improves RTT phenotypes. Our study thus provides a much-needed female model and implies potential therapeutic benefit with low-level MECP2 expression.

Tsix–Mecp2 female mouse model for Rett syndrome reveals that low-level MECP2 expression extends life and improves neuromotor function