title: The promising trajectory of autism therapeutics discovery.
abstract: Pharmacological interventions for neurodevelopmental disorders are increasingly tractable. Autism is a neurodevelopmental disorder that affects approximately 1% of the population. Currently, the standard of care is early behavioral therapy. No approved medical treatments for the diagnostic symptoms are available. Strong evidence for genetic causes of autism implicates proteins that mediate synaptic transmission and structure. Mouse models with targeted mutations in these synaptic genes display behavioral symptoms relevant to the social communication abnormalities and repetitive behaviors that define autism spectrum disorder (ASD), along with biological abnormalities in synaptic physiology and morphology. As we discuss here, promising pharmacological targets, emerging from the mouse model studies, are now being pursued in early clinical trials. Thus, a high-prevalence disorder that was previously considered to be medically untreatable is now moving into the therapeutic arena.
authors.affiliation: MIND Institute, Department of Psychiatry and Behavioral Sciences, University of California Davis School of Medicine, Sacramento, CA 95817, USA.
authors.forename: Jill L
authors.initials: JL
authors.lastname: Silverman
authors.affiliation: MIND Institute, Department of Psychiatry and Behavioral Sciences, University of California Davis School of Medicine, Sacramento, CA 95817, USA. Electronic address: jacqueline.crawley@ucdmc.ucdavis.edu.
authors.forename: Jacqueline N
authors.initials: JN
authors.lastname: Crawley
mesh_terms: D000818:Animals; D001321:Autistic Disorder / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics*; D055808:Drug Discovery / Q000379:methods / Q000639:trends*; D018683:Excitatory Amino Acid Agents / Q000627:therapeutic use; D018757:GABA Modulators / Q000627:therapeutic use; D020022:Genetic Predisposition to Disease / Q000235:genetics*; D006801:Humans
chemical_list: D018683:Excitatory Amino Acid Agents; D018757:GABA Modulators