Targeted pharmacological treatment of autism spectrum disorders: fragile X and Rett syndromes

Frontiers in Cellular Neuroscience
Hansen WangLaurie C Doering

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.

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Citations

Jun 2, 2015·Frontiers in Cellular Neuroscience·Hansen Wang, Laurie C Doering
Oct 19, 2016·Human Gene Therapy·Jason ArsenaultDavid R Hampson
Jan 26, 2017·Proceedings of the National Academy of Sciences of the United States of America·Alicia MansillaMaría José Sánchez-Barrena
Mar 25, 2017·Frontiers in Molecular Neuroscience·Steffen VogelgesangMarcus Niebert
Jun 26, 2018·Frontiers in Synaptic Neuroscience·Hansen Wang
Feb 7, 2017·Expert Opinion on Orphan Drugs·Walter E KaufmannSteven A Skinner
Sep 14, 2017·Developmental Neurobiology·Joshua M Lorenz-Guertin, Tija C Jacob
Nov 20, 2020·American Journal on Intellectual and Developmental Disabilities·Melissa RaspaJeffrey L Neul
Jan 3, 2018·Journal of Developmental and Behavioral Pediatrics : JDBP·Adam BraddockMartin T Stein
Feb 9, 2017·Future Medicinal Chemistry·Hendra GunosewoyoMichael Kassiou

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Methods Mentioned

BETA
pharmacotherapy
GTPase
pharmacotherapies

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