Translating genetic and preclinical findings into autism therapies

Authors

Maria Chahrour
Robin Kleiman
M Chiara Manzini, George Washington UniversityFollow

Document Type

Journal Article

Publication Date

12-2017

Journal

Dialogues in Clinical Neuroscience

Volume

19

Issue

4

Inclusive Pages

335-343

Abstract

Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder characterized by social deficits and repetitive/restrictive interests. ASD is associated with multiple comorbidities, including intellectual disability, anxiety, and epilepsy. Evidence that ASD is highly heritable has spurred major efforts to unravel its genetics, revealing possible contributions from hundreds of genes through rare and common variation and through copy-number changes. In this perspective, we provide an overview of the current state of ASD genetics and of how genetic research has spurred the development of in vivo and in vitro models using animals and patient cells to evaluate the impact of genetic mutations on cellular function leading to disease. Efforts to translate these findings into successful therapies have yet to bear fruit. We discuss how the valuable insight into the disorder provided by these new models can be used to better understand ASD and develop future clinical trials.

Comments

Copyright : © 2017 AICH - Servier Research Group. All rights reserved

Creative Commons License

This work is licensed under a Creative Commons Attribution 3.0 License.

APA Citation

Chahrour, M., Kleiman, R., & Manzini, M. (2017). Translating genetic and preclinical findings into autism therapies. Dialogues in Clinical Neuroscience, 19 (4). Retrieved from https://hsrc.himmelfarb.gwu.edu/smhs_pharm_facpubs/209

Open Access

1