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...
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American Association for the Advancement of Science (AAAS)
2016
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Online Access: | http://hdl.handle.net/1721.1/103768 https://orcid.org/0000-0003-2442-5671 |
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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) |
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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 |
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