Neurometabolic Diseases Lab

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Home Category Table Improvement of the Rett Syndrome Phenotype in a Mecp2 Mouse Model Upon Treatment with Levodopa and a Dopa Decarboxylase Inhibitor. Neuropsychopharmacology. 2014 Jun 11. doi: 10.1038/npp.2014.136. [Epub ahead of print]
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Szczesna K, de la Caridad O, Petazzi P, Soler M, Roa L, Saez MA, Fourcade S, Pujol A, Artuch-Iriberri R, Molero-Luis M, Vidal A, Huertas D, Esteller M. Improvement of the Rett Syndrome Phenotype in a Mecp2 Mouse Model Upon Treatment with Levodopa and a Dopa Decarboxylase Inhibitor. Neuropsychopharmacology. 2014 Jun 11. doi: 10.1038/npp.2014.136. [Epub ahead of print].

Rett Syndrome is a neurodevelopmental autism spectrum disorder caused by mutations in the gene coding for methyl CpG-binding protein (MeCP2). The disease is characterized by abnormal motor, respiratory, cognitive impairment, and autistic-like behaviors. No effective treatment of the disorder is available. Mecp2 knockout mice have a range of physiological and neurological abnormalities that resemble the human syndrome and can be used as a model to interrogate new therapies. Herein, we show that the combined administration of Levodopa and a Dopa decarboxylase inhibitor in Rett syndrome mouse models is well tolerated, diminishes Rett syndrome-associated symptoms and increases lifespan. The amelioration of Rett syndrome symptomatology is particularly significant in those features controlled by the dopaminergic pathway in the nigrostratium, such as mobility, tremor and breathing. Most important, the improvement of the Rett syndrome phenotype upon use of the combined treatment is reflected at the cellular level by the development of neuronal dendritic growth. However, much work is required to extend the duration of the benefit of the described preclinical treatment.Neuropsychopharmacology accepted article preview online, 11 June 2014; doi:10.1038/npp.2014.136.

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Last Updated on Tuesday, 01 July 2014 08:55  

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