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

Rett syndrome 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 conditio...

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Detalles Bibliográficos
Autores principales: Luoni, Mirko, Giannelli, Serena, Indrigo, Marzia Tina, Niro, Antonio, Massimino, Luca, Iannielli, Angelo, Passeri, Laura, Russo, Fabio, Morabito, Giuseppe, Calamita, Piera, Gregori, Silvia, Deverman, Benjamin, Broccoli, Vania
Formato: Online Artículo Texto
Lenguaje:English
Publicado: eLife Sciences Publications, Ltd 2020
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7117907/
https://www.ncbi.nlm.nih.gov/pubmed/32207685
http://dx.doi.org/10.7554/eLife.52629

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