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 aut-ism spectrum disorders (ASD) and schizophrenia (SCZ). Clinical treatment of these disorders has tradi-tionally 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 molec-ular 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 treat-ment and discuss ongoing preclinical efforts as well as new initiatives aimed at developing scalable plat-forms for NDD precision medicine. (c) 2022 The Author(s). Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

Automated summary

Neurodevelopmental Disorders (NDDs) encompass a wide range of disorders, including autism spectrum disorders (ASD) and schizophrenia (SCZ), which are linked to brain developmental processes. The clinical treatment of these disorders has traditionally focused on symptom management due to the variability in developmental disruption and limited knowledge of the underlying molecular mechanisms. With the identification of several hundred genes associated with ASD and SCZ, there is potential for new therapeutic approaches and the opportunity for precision medicine. This review discusses the progress in research technologies that enhance our understanding of disease progression and highlights advances in targeted clinical treatment and ongoing preclinical efforts in NDD precision medicine.