Genes Dysregulated in Autism as Potential Biomarkers and Therapeutic Targets

University of California System: University of California, Los Angeles - UCLA
posted on 05/02/2012

Brief description unavailable


  • Identifies a cohort of gene expression changes in the disease-relevant tissue (ASD brain).
  • Can be used as genetic biomarkers for the accurate and early diagnosis of ASD in young children.
  • Describes the dysregulated molecular pathways that may include candidates for preventive approaches or treatment therapeutics for ASD patients.
  • Some of the gene changes are also seen in peripheral blood lymphocytes, indicating that they may serve as biomarkers for ASD diagnosis.

Innovation Details

Detailed Description

Researchers at UCLA have characterized the genome-wide expression profiles of postmortem brain tissue from several brain regions in ASD patients and controls. They identified numerous genes showing consistent changes in expression level and employed a network-based approach to identify groups of functionally-related genes that are aberrantly expressed in the ASD brain. These analyses highlight genes that are dysregulated in ASD in the disease-relevant tissue, and define a set of co-expressed genes that may serve as therapeutic candidates or potential biomarkers for the disease. Some of the identified genes also change in peripheral blood lymphocytes, suggesting that these genes may serve as diagnostic biomarkers in peripheral tissue samples. The molecular entities identified herein can be utilized as a genetic diagnostic test for ASD, or exploited for the discovery and development of ASD therapeutics. 

File Number: 22469 

Disease: Central Nervous System

Other Information:


In the United States, autism spectrum disorder (ASD) affects an estimated one in 110 children, and its prevalence continues to increase. The lack of effective means of genetic diagnosis, prevention, and treatment emphasizes the importance of identifying biomarkers and molecular pathways implicated in ASD. Most studies of gene expression in ASD have profiled only peripheral tissues, which show expression patterns that largely differ from that of brain tissue. Gene expression changes in the ASD brain may serve as relevant biomarkers for diagnosis, or as therapeutic targets for disease treatment.

IP Protection

Patent Number(s): 2014/0194310
Copyright: ©2012-2014, The Regents of the University of California

License Online

This innovation currently is not available for online licensing. Please contact UCLA Office of Intellectual Property & Industry Sponsored Research at University of California System: University of California, Los Angeles - UCLA for more information.

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