In vivo repair of a protein underlying a neurological disorder by programmable RNA editing

The goal of this study is to determine the effects of targeted RNA editing in the hippocampus of a mouse model of Rett syndrome. Mice containing a patient G>A mutation in Methyl CpG binding Protein 2 (MeCP2) have been injected with AAV expressing an engineered deaminase with a guide targeting Mecp2 RNA or a non-targeting guide.