title: Targeted pharmacological treatment of autism spectrum disorders: fragile X and Rett syndromes.
abstract: Autism spectrum disorders (ASDs) are genetically and clinically heterogeneous and lack effective medications to treat their core symptoms. Studies of syndromic ASDs caused by single gene mutations have provided insights into the pathophysiology of autism. Fragile X and Rett syndromes belong to the syndromic ASDs in which preclinical studies have identified rational targets for drug therapies focused on correcting underlying neural dysfunction. These preclinical discoveries are increasingly translating into exciting human clinical trials. Since there are significant molecular and neurobiological overlaps among ASDs, targeted treatments developed for fragile X and Rett syndromes may be helpful for autism of different etiologies. Here, we review the targeted pharmacological treatment of fragile X and Rett syndromes and discuss related issues in both preclinical studies and clinical trials of potential therapies for the diseases.
authors.affiliation: Faculty of Medicine, University of Toronto, 1 King's College Circle Toronto, ON, Canada.
authors.forename: Hansen
authors.initials: H
authors.lastname: Wang
authors.affiliation: Department of Neurology, Epilepsy Division, The University of Alabama at Birmingham Birmingham, AL, USA.
authors.forename: Sandipan
authors.initials: S
authors.lastname: Pati
authors.affiliation: Department of Neurobiology, Civitan International Research Center, The University of Alabama at Birmingham Birmingham, AL, USA.
authors.forename: Lucas
authors.initials: L
authors.lastname: Pozzo-Miller
authors.affiliation: Faculty of Health Sciences, Department of Pathology and Molecular Medicine, McMaster University Hamilton, ON, Canada.
authors.forename: Laurie C
authors.initials: LC
authors.lastname: Doering
keywords: FMRP; MeCP2; Rett syndrome; autism spectrum disorders; fragile X syndrome; pharmacotherapy; synaptic deficits; treatment