Neurobiology: Domains to the rescue for Rett syndrome.

Abstract

Rett syndrome is a brain disorder caused by disrupted forms of the protein MECP2, but how MECP2 loss affects the brain is unknown. A mouse study now implicates key domains of the protein and offers therapeutic insights. See Letter p.398 Rett syndrome is a neurological disorder caused by mutations in the MECP2 gene, which tend to be clustered in two discrete regions of the protein (MeCP2). In this report, the authors parse the minimal form of MeCP2 that is required to retain its functionality, and interrogate which of its many proposed roles is relevant for Rett syndrome progression. The identification of a minimal functional unit for MeCP2 could be helpful in the design of therapeutic strategies for gene therapy for Rett syndrome.