Improvement of the Rett syndrome phenotype in a MeCP2 mouse model upon treatment with levodopa and a dopa-decarboxylase inhibitor

Neuropsychopharmacology. 2014 Nov;39(12):2846-56. doi: 10.1038/npp.2014.136. Epub 2014 Jun 11.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Anti-Dyskinesia Agents / pharmacology*
  • Aromatic Amino Acid Decarboxylase Inhibitors / pharmacology*
  • Body Weight / drug effects
  • Brain / drug effects
  • Brain / pathology
  • Brain / physiopathology
  • Cell Enlargement / drug effects
  • Dendrites / drug effects
  • Dendrites / pathology
  • Dendrites / physiology
  • Disease Models, Animal
  • Dopa Decarboxylase / metabolism
  • Dopaminergic Neurons / drug effects
  • Dopaminergic Neurons / pathology
  • Dopaminergic Neurons / physiology
  • Levodopa / pharmacology*
  • Male
  • Methyl-CpG-Binding Protein 2 / deficiency*
  • Methyl-CpG-Binding Protein 2 / genetics
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Movement / drug effects
  • Phenotype
  • Respiration / drug effects
  • Rett Syndrome / drug therapy*
  • Rett Syndrome / pathology
  • Rett Syndrome / physiopathology

Substances

  • Anti-Dyskinesia Agents
  • Aromatic Amino Acid Decarboxylase Inhibitors
  • Mecp2 protein, mouse
  • Methyl-CpG-Binding Protein 2
  • Levodopa
  • Dopa Decarboxylase