期刊
NEUROPSYCHOPHARMACOLOGY
卷 39, 期 12, 页码 2846-2856出版社
SPRINGERNATURE
DOI: 10.1038/npp.2014.136
关键词
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资金
- European Community [PITN-GA-2012-316758- EPITRAIN, PITN-GA-2009-238242-DISCHROM]
- ERC [268626-EPINORC]
- E-RARE EuroRETT network [PI071327]
- GIBER on Rare Diseases (CIBERER) from the Carlos III Health Institute
- Fondation Lejeune (France)
- MINECO [SAF2011-22803, CSD2006-00049]
- Cellex Foundation
- Botin Foundation
- Catalan Association for Rett Syndrome
- Health and Science Departments of the Catalan Government (Generalitat de Catalunya) [AGAUR 2009SGR1315, 2009SGR85]
- ICREA Funding Source: Custom
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 RTT mouse models is well tolerated, diminishes RTT-associated symptoms, and increases life span. The amelioration of RTT 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 RTT 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.
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