MeCP2-boosting Approaches Led to Toxic Events in Mice

MeCP2-boosting Approaches Led to Toxic Events in Mice

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MeCP2-boosting Approaches Led to Toxic Events in Mice

Providing a working version of MECP2 — the gene mutated in most cases of Rett syndrome — to cells reversed disease-specific symptoms in a mouse model of a common, milder form of Rett syndrome, a study shows. However, female mice treated with this approach showed signs of excessive, toxic levels of MeCP2, the protein coded by the MECP2 gene and missing or defective in Rett patients. These findings add to increasing evidence pointing out the fine balance of protein levels that MeCP2-boosting strategies,…

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