Article
Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome.
eLife - 24 Mar 2020
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
Abstract excerpt
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 conditions as well. Herein, we use the AAV-PHP.eB to deliver an instability-prone Mecp2 (iMecp2) transgene cassette which,...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.