Antipurinergic therapy corrects the autism-like features in the Fragile X (Fmr1 knockout) mouse model.

ABSTRACT:

Background

This study was designed to test a new approach to drug treatment of autism spectrum disorders (ASDs) in the Fragile X (Fmr1) knockout mouse model.

Methods

We used behavioral analysis, mass spectrometry, metabolomics, electron microscopy, and western analysis to test the hypothesis that the disturbances in social behavior, novelty preference, metabolism, and synapse structure are treatable with antipurinergic therapy (APT).

Results

Weekly treatment with the purinergic antagonist suramin (20 mg/kg intraperitoneally), started at 9 weeks of age, restored normal social behavior, and improved metabolism, and brain synaptosomal structure. Abnormalities in synaptosomal glutamate, endocannabinoid, purinergic, and IP3 receptor expression, complement C1q, TDP43, and a

SUBMITTER: Naviaux JC 

PROVIDER: S-EPMC4334917 | biostudies-literature | 2015

REPOSITORIES: biostudies-literature