Improvement of the Rett Syndrome Phenotype in a Mecp2 Mouse Model Upon Treatment with Levodopa and a Dopa-Decarboxylase Inhibitor

• DOI: 10.1038/npp.2014.136
• Publication Date: 2014-06-11T10:58:27Z
• Authors: Karolina Szczesna; Olga de la Caridad; Paolo Petazzi; Marta Soler; Laura Roa; Mauricio A Saez; Stéphane Fourcade; Aurora Pujol; Rafael Artuch-Iri...; Marta Molero-Luis; August Vidal; Dori Huertas; Manel Esteller
• Keywords: Rett Syndrome; MeCP2; Neurodevelopmental disorder

Rett Syndrome is a neurodevelopmental autism spectrum disorder caused by mutations in the gene coding for methyl CpG-binding protein (MeCP2). The disease characterized abnormal motor, respiratory, cognitive impairment, and autistic-like behaviors. No effective treatment of available. Mecp2 knockout mice have range physiological neurological abnormalities that resemble human syndrome can be used as model to interrogate new therapies. Herein, we show combined administration Levodopa Dopa-decarboxylase inhibitor RTT mouse models well tolerated, diminishes RTT-associated symptoms, increases life span. amelioration symptomatology particularly significant those features controlled dopaminergic pathway nigrostratium, such mobility, tremor, breathing. Most important, improvement phenotype upon use reflected at cellular level development neuronal dendritic growth. However, much work required extend duration benefit described preclinical treatment.