Could Autism Be Treated Prenatally?

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Science  07 Feb 2014:
Vol. 343, Issue 6171, pp. 620-621
DOI: 10.1126/science.1250214

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Autism spectrum disorder (ASD) has presented a conundrum: How can the behavioral signs and symptoms that define the condition arise from different etiologies and lead to so many varied phenotypes? Genetics and the environment, including prenatal and perinatal factors, long have been suspected to interact in the causation of ASD. Evidence for neuronal dysfunction and the frequent development of epilepsy strongly support increased excitatory and decreased inhibitory neuronal activity in ASD. In particular, altered functions of γ-aminobutyric acid (GABA), the main inhibitory neurotransmitter (in the mature brain), have been of interest because GABA's effects are excitatory during prenatal development but become inhibitory at birth (1, 2). What has been unclear is the cellular physiology that underlies this “GABA switch.” On page 675 of this issue, Tyzio et al. (3) show that a defect in this switch is associated with abnormal chloride concentration in neurons in two different animal models of ASD. Normal electrophysiology and behavior can be restored in their offspring by the prenatal administration of the compound bumetanide, which blocks a key chloride transporter. The findings raise the possibility of preventing the autistic phenotype in offspring by predelivery pharmacological treatment.