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Intractable Rare Dis Res. 2014 Nov;3(4):110-7. doi: 10.5582/irdr.2014.01027.

Serotonin dysregulation in Fragile X Syndrome: implications for treatment.

Intractable & rare diseases research

Alicia C Hanson, Randi J Hagerman

Affiliations

  1. UC Davis School of Medicine, Sacramento, CA, USA;
  2. UC Davis MIND Institute and Department of Pediatrics, UC Davis Medical Center, Sacramento, CA, USA.

PMID: 25606361 PMCID: PMC4298641 DOI: 10.5582/irdr.2014.01027

Abstract

Fragile X Syndrome (FXS) is a trinucleotide repeat disorder that results in the silencing of the Fragile X Mental Retardation 1 gene (FMR1), leading to a lack of the FMR1 protein (FMRP). FMRP is an mRNA-binding protein that regulates the translation of hundreds of mRNAs important for synaptic plasticity. Several of these pathways have been identified and have guided the development of targeted treatments for FXS. Here we present evidence that serotonin is dysregulated in FXS and treatment with the selective serotonin reuptake inhibitor (SSRI) sertraline may be beneficial for individuals with FXS, particularly in early childhood.

Keywords: Fragile X Syndrome; fragile X mental retardation protein; selective serotonin reuptake inhibitors; sertraline

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