Article

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

2020-02-27

Abstract excerpt

RNA base editing is gaining momentum as an approach to repair mutations, but its application to neurological disease has not been established. We have succeeded in directed transcript editing of a pathological mutation in a mouse model of the neurodevelopmental disease, Rett syndrome. Specifically, we directed editing of a guanosine to adenosine mutation in RNA encoding Methyl CpG Binding Protein 2 (MECP2). Repair...

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Literature Corpus work: 6f6743af-32a5-5c81-8292-6f05aad3acc7
DOI: 10.1101/2020.02.26.966820

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