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Challenges and opportunities for precision medicine in neurodevelopmental disorders

Neurodevelopmental Disorders (NDDs) encompass a broad spectrum of disorders, linked because of their origins in brain developmental processes, including diverse conditions across the age span, including autism spectrum disorders (ASD) and schizophrenia (SCZ). Clinical treatment of these disorders ha...

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Detalles Bibliográficos
Autores principales: Chen, George T., Geschwind, Daniel H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10409256/
https://www.ncbi.nlm.nih.gov/pubmed/36183905
http://dx.doi.org/10.1016/j.addr.2022.114564
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author Chen, George T.
Geschwind, Daniel H.
author_facet Chen, George T.
Geschwind, Daniel H.
author_sort Chen, George T.
collection PubMed
description Neurodevelopmental Disorders (NDDs) encompass a broad spectrum of disorders, linked because of their origins in brain developmental processes, including diverse conditions across the age span, including autism spectrum disorders (ASD) and schizophrenia (SCZ). Clinical treatment of these disorders has traditionally focused on symptom management, as the severity of developmental disruption varies widely and the precise molecular mechanisms, timing, and progression of these disorders is usually not known. Several hundred genes have been identified as major risk factors for ASD and SCZ, which creates new potential therapeutic avenues, and there is strong evidence that these genes converge upon key molecular pathways, pointing to opportunities for precision medicine. In this review, we focus on forms of ASD and SCZ with known genetic etiologies and discuss advances in research technologies that enable a more systemic understanding of disease progression. We highlight recent advances in targeted clinical treatment and discuss ongoing preclinical efforts as well as new initiatives aimed at developing scalable platforms for NDD precision medicine.
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spelling pubmed-104092562023-08-08 Challenges and opportunities for precision medicine in neurodevelopmental disorders Chen, George T. Geschwind, Daniel H. Adv Drug Deliv Rev Article Neurodevelopmental Disorders (NDDs) encompass a broad spectrum of disorders, linked because of their origins in brain developmental processes, including diverse conditions across the age span, including autism spectrum disorders (ASD) and schizophrenia (SCZ). Clinical treatment of these disorders has traditionally focused on symptom management, as the severity of developmental disruption varies widely and the precise molecular mechanisms, timing, and progression of these disorders is usually not known. Several hundred genes have been identified as major risk factors for ASD and SCZ, which creates new potential therapeutic avenues, and there is strong evidence that these genes converge upon key molecular pathways, pointing to opportunities for precision medicine. In this review, we focus on forms of ASD and SCZ with known genetic etiologies and discuss advances in research technologies that enable a more systemic understanding of disease progression. We highlight recent advances in targeted clinical treatment and discuss ongoing preclinical efforts as well as new initiatives aimed at developing scalable platforms for NDD precision medicine. 2022-12 2022-09-29 /pmc/articles/PMC10409256/ /pubmed/36183905 http://dx.doi.org/10.1016/j.addr.2022.114564 Text en https://creativecommons.org/licenses/by/4.0/This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) ).
spellingShingle Article
Chen, George T.
Geschwind, Daniel H.
Challenges and opportunities for precision medicine in neurodevelopmental disorders
title Challenges and opportunities for precision medicine in neurodevelopmental disorders
title_full Challenges and opportunities for precision medicine in neurodevelopmental disorders
title_fullStr Challenges and opportunities for precision medicine in neurodevelopmental disorders
title_full_unstemmed Challenges and opportunities for precision medicine in neurodevelopmental disorders
title_short Challenges and opportunities for precision medicine in neurodevelopmental disorders
title_sort challenges and opportunities for precision medicine in neurodevelopmental disorders
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10409256/
https://www.ncbi.nlm.nih.gov/pubmed/36183905
http://dx.doi.org/10.1016/j.addr.2022.114564
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