Autism Spectrum Disorders Buxbaum, Joseph D.; Kolevzon, Alexander; Wang, A. Ting ...
Neurobiology of Mental Illness,
07/2013
Book Chapter
Autism spectrum disorders (ASD) manifest with deficits in social communication, together with the presence of repetitive behaviors and restricted interests.Behavioral interventions are important ...firstline treatments in ASD and there is a need for pharmacological treatments that target core symptoms of ASD. Neuroimaging studies implicate specific brain regions and networks in ASD, and there has beensignificant progress in identifying etiological genetic loci for ASD. Gene discovery, coupled with the development of animal model systems, have elucidated the neurobiology of several monogenic forms of ASD. These discoveries have in turn led to important opportunities for developing novel treatments and several clinical trials are underway with potentially diseasemodifying medications that target core symptom domains. Although the state of the evidence is preliminary, these studies are the basis for enormous optimism in ASD and underscore how neurobiological approaches are likely to lead to novel therapeutics in ASD.
Neurodevelopmental disorders are frequently the result of genetic and genomic abnormalities associated with high risk for disease. Creating analogous mutations in cell and animal models permits the ...assessment of underlying neurobiological mechanisms, generates clues about useful therapeutic targets, and provides systems for preclinical evaluation of novel therapeutics. This chapter briefly summarizes several clinical trials in neurodevelopmental disorders, all based on neurobiological findings in model systems, including trials in Down syndrome (DS) and several monogenic forms of intellectual disability (ID) and/or autism spectrum disorder (ASD).
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