Cellular, electrophysiological and behavioral improvements in a mouse model of Rett syndrome following gene therapy combined with focused ultrasound-mediated blood-brain barrier opening

Authors
Marie-Solenne Felix, Léna Bourcin, Emilie Borloz, Khaled Metwally, Benoit Larrat, Serge Mensah, Yasmine Belaidouni, Jean-Luc Gaiarsa, Anthony Novell, Laurent Villard, Jean-Christophe Roux


Lab

Journal
Experimental Neurology

Abstract
Rett syndrome (RTT) is a severe X-linked neurodevelopmental disorder due to pathogenic variants in the methyl CpG binding protein 2 gene (MECP2). The

Keywords/Topics
electrophysiological and behavioral improvements; each mouse; rett syndrome; gene therapy; focused ultrasound-mediated blood-brain barrier; pathogenic variants; mecp2; panlab; forelimb strength; five measures

Source :

https://www.sciencedirect.com/science/article/pii/S0014488626002736

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