Article

Whole brain delivery of an instability-prone <i>Mecp2</i> transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome

2019-10-09

Abstract excerpt

Rett syndrome (RTT) is an incurable neurodevelopmental disorder caused by mutations in the gene encoding for methyl-CpG binding-protein 2 (MeCP2). Gene therapy for this disease presents inherent hurdles since MECP2 is expressed throughout the brain and its duplication leads to severe neurological conditions as well. However, the recent introduction of AAV-PHP.eB, an engineered capsid with an unprecedented efficie...

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Literature Corpus work: 3da1438f-4a88-54ea-b3ff-6d5cf1a0680e
DOI: 10.1101/798793

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