A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome

A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome

Rett syndrome (RTT) is a severe X-linked neurodevelopmental disorder that is primarily caused by mutations in the methyl CpG binding protein 2 gene (MECP2). RTT is the second most prevalent cause of intellectual disability in girls and there is currently no cure for the disease. The finding that the...

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Bibliographic Details
Main Authors: Valerie Matagne, Yann Ehinger, Lydia Saidi, Ana Borges-Correia, Martine Barkats, Marc Bartoli, Laurent Villard, Jean-Christophe Roux
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Neurobiology of Disease
Subjects:
Rett syndrome
Mecp2
AAV9
Gene therapy
Animal model
Online Access:http://www.sciencedirect.com/science/article/pii/S096999611630290X

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