Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders

Research in the genetics of neurodevelopmental disorders such as autism suggests that several hundred genes are likely risk factors for these disorders. This heterogeneity presents a challenge and an opportunity at the same time. Although the exact identity of many of the genes remains to be discove...

Full description

Bibliographic Details
Main Authors: Sahin, Mustafa, Sur, Mriganka
Other Authors: Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences
Format: Article
Language:en_US
Published: American Association for the Advancement of Science (AAAS) 2016
Online Access:http://hdl.handle.net/1721.1/103768
https://orcid.org/0000-0003-2442-5671
_version_ 1826205718992125952
author Sahin, Mustafa
Sur, Mriganka
author2 Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences
author_facet Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences
Sahin, Mustafa
Sur, Mriganka
author_sort Sahin, Mustafa
collection MIT
description Research in the genetics of neurodevelopmental disorders such as autism suggests that several hundred genes are likely risk factors for these disorders. This heterogeneity presents a challenge and an opportunity at the same time. Although the exact identity of many of the genes remains to be discovered, genes identified to date encode proteins that play roles in certain conserved pathways: protein synthesis, transcriptional and epigenetic regulation, and synaptic signaling. The next generation of research in neurodevelopmental disorders must address the neural circuitry underlying the behavioral symptoms and comorbidities, the cell types playing critical roles in these circuits, and common intercellular signaling pathways that link diverse genes. Results from clinical trials have been mixed so far. Only when we can leverage the heterogeneity of neurodevelopmental disorders into precision medicine will the mechanism-based therapeutics for these disorders start to unlock success.
first_indexed 2024-09-23T13:17:30Z
format Article
id mit-1721.1/103768
institution Massachusetts Institute of Technology
language en_US
last_indexed 2024-09-23T13:17:30Z
publishDate 2016
publisher American Association for the Advancement of Science (AAAS)
record_format dspace
spelling mit-1721.1/1037682022-09-28T13:14:54Z Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders Sahin, Mustafa Sur, Mriganka Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences Picower Institute for Learning and Memory Sur, Mriganka Research in the genetics of neurodevelopmental disorders such as autism suggests that several hundred genes are likely risk factors for these disorders. This heterogeneity presents a challenge and an opportunity at the same time. Although the exact identity of many of the genes remains to be discovered, genes identified to date encode proteins that play roles in certain conserved pathways: protein synthesis, transcriptional and epigenetic regulation, and synaptic signaling. The next generation of research in neurodevelopmental disorders must address the neural circuitry underlying the behavioral symptoms and comorbidities, the cell types playing critical roles in these circuits, and common intercellular signaling pathways that link diverse genes. Results from clinical trials have been mixed so far. Only when we can leverage the heterogeneity of neurodevelopmental disorders into precision medicine will the mechanism-based therapeutics for these disorders start to unlock success. National Institutes of Health (U.S.) (NIH grant MH085802) Simons Foundation (Autism Research Initiative) National Institutes of Health (U.S.) (NIH grant U01 NS082320) National Institutes of Health (U.S.) (NIH grant P20 NS080199) National Institutes of Health (U.S.) (NIH grant P30 HD018655) National Institutes of Health (U.S.) (NIH grant EY007023) United States. Dept. of Defense (W81XWH-13-1-0040) United States. Dept. of Defense (W81XWH-15-1-0189) Simons Foundation Nancy Lurie Marks Family Foundation Boston Children's Hospital (Translational Research Program) Autism Speaks (Organization) Tuberous Sclerosis Alliance National Institutes of Health (U.S.). Office of Rare Diseases Research (Developmental Synaptopathies Consortium (U54NS092090)) 2016-07-20T14:14:38Z 2016-07-20T14:14:38Z 2016-07-20 Article http://purl.org/eprint/type/JournalArticle 0036-8075 1095-9203 http://hdl.handle.net/1721.1/103768 Sahin, Mustafa, and Mriganka Sur. “Genes, Circuits, and Precision Therapies for Autism and Related Neurodevelopmental Disorders.” Science 350, no. 6263 (October 15, 2015): aab3897–aab3897. https://orcid.org/0000-0003-2442-5671 en_US http://dx.doi.org/10.1126/science.aab3897 Science Creative Commons Attribution-Noncommercial-Share Alike http://creativecommons.org/licenses/by-nc-sa/4.0/ application/pdf American Association for the Advancement of Science (AAAS) PMC
spellingShingle Sahin, Mustafa
Sur, Mriganka
Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title_full Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title_fullStr Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title_full_unstemmed Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title_short Genes, circuits, and precision therapies for autism and related neurodevelopmental disorders
title_sort genes circuits and precision therapies for autism and related neurodevelopmental disorders
url http://hdl.handle.net/1721.1/103768
https://orcid.org/0000-0003-2442-5671
work_keys_str_mv AT sahinmustafa genescircuitsandprecisiontherapiesforautismandrelatedneurodevelopmentaldisorders
AT surmriganka genescircuitsandprecisiontherapiesforautismandrelatedneurodevelopmentaldisorders