D-cycloserine improves synaptic transmission in an animal model of Rett syndrome

PLoS One. 2017 Aug 16;12(8):e0183026. doi: 10.1371/journal.pone.0183026. eCollection 2017.

Abstract

Rett syndrome (RTT), a leading cause of intellectual disability in girls, is predominantly caused by mutations in the X-linked gene MECP2. Disruption of Mecp2 in mice recapitulates major features of RTT, including neurobehavioral abnormalities, which can be reversed by re-expression of normal Mecp2. Thus, there is reason to believe that RTT could be amenable to therapeutic intervention throughout the lifespan of patients after the onset of symptoms. A common feature underlying neuropsychiatric disorders, including RTT, is altered synaptic function in the brain. Here, we show that Mecp2tm1.1Jae/y mice display lower presynaptic function as assessed by paired pulse ratio, as well as decreased long term potentiation (LTP) at hippocampal Schaffer-collateral-CA1 synapses. Treatment of Mecp2tm1.1Jae/y mice with D-cycloserine (DCS), an FDA-approved analog of the amino acid D-alanine with antibiotic and glycinergic activity, corrected the presynaptic but not LTP deficit without affecting deficient hippocampal BDNF levels. DCS treatment did, however, partially restore lower BDNF levels in the brain stem and striatum. Thus, treatment with DCS may mitigate the severity of some of the neurobehavioral symptoms experienced by patients with Rett syndrome.

MeSH terms

  • Animals
  • Apnea
  • Brain Stem / metabolism
  • Brain Stem / physiopathology
  • Brain-Derived Neurotrophic Factor / metabolism
  • Corpus Striatum / metabolism
  • Corpus Striatum / physiopathology
  • Cycloserine / administration & dosage
  • Cycloserine / pharmacology*
  • Disease Models, Animal
  • Gait / drug effects
  • Hippocampus / drug effects
  • Hippocampus / physiopathology
  • Locomotion / drug effects
  • Male
  • Methyl-CpG-Binding Protein 2 / genetics
  • Mice
  • Mice, Transgenic
  • Muscle Strength / drug effects
  • Rett Syndrome / drug therapy
  • Rett Syndrome / genetics
  • Rett Syndrome / metabolism
  • Rett Syndrome / physiopathology*
  • Synaptic Transmission / drug effects*
  • Tremor

Substances

  • Brain-Derived Neurotrophic Factor
  • Methyl-CpG-Binding Protein 2
  • Cycloserine