Genomics, convergent neuroscience and progress in understanding autism spectrum disorder

HR Willsey, AJ Willsey, B Wang… - Nature Reviews …, 2022 - nature.com
More than a hundred genes have been identified that, when disrupted, impart large risk for
autism spectrum disorder (ASD). Current knowledge about the encoded proteins—although …

Advancing the understanding of autism disease mechanisms through genetics

L de la Torre-Ubieta, H Won, JL Stein… - Nature medicine, 2016 - nature.com
Progress in understanding the genetic etiology of autism spectrum disorders (ASD) has
fueled remarkable advances in our understanding of its potential neurobiological …

Optical genome mapping enables constitutional chromosomal aberration detection

T Mantere, K Neveling, C Pebrel-Richard… - The American Journal of …, 2021 - cell.com
Chromosomal aberrations including structural variations (SVs) are a major cause of human
genetic diseases. Their detection in clinical routine still relies on standard cytogenetics …

Autism genetics: opportunities and challenges for clinical translation

JAS Vorstman, JR Parr, D Moreno-De-Luca… - Nature Reviews …, 2017 - nature.com
Genetic studies have revealed the involvement of hundreds of gene variants in autism. Their
risk effects are highly variable, and they are frequently related to other conditions besides …

Down syndrome and the complexity of genome dosage imbalance

SE Antonarakis - Nature Reviews Genetics, 2017 - nature.com
Down syndrome (also known as trisomy 21) is the model human phenotype for all genomic
gain dosage imbalances, including microduplications. The functional genomic exploration of …

Parallel in vivo analysis of large-effect autism genes implicates cortical neurogenesis and estrogen in risk and resilience

HR Willsey, CRT Exner, Y Xu, A Everitt, N Sun, B Wang… - Neuron, 2021 - cell.com
Gene Ontology analyses of autism spectrum disorders (ASD) risk genes have repeatedly
highlighted synaptic function and transcriptional regulation as key points of convergence …

Syndromic autism spectrum disorders: moving from a clinically defined to a molecularly defined approach

BA Fernandez, SW Scherer - Dialogues in clinical neuroscience, 2017 - Taylor & Francis
Autism spectrum disorder (ASD) encompasses a group of neurodevelopmental conditions
diagnosed solely on the basis of behavioral assessments that reveal social deficits …

Dual-specificity, tyrosine phosphorylation-regulated kinases (DYRKs) and cdc2-like kinases (CLKs) in human disease, an overview

MF Lindberg, L Meijer - International Journal of Molecular Sciences, 2021 - mdpi.com
Dual-specificity tyrosine phosphorylation-regulated kinases (DYRK1A, 1B, 2-4) and cdc2-
like kinases (CLK1-4) belong to the CMGC group of serine/threonine kinases. These protein …

Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

C De Masi, P Spitalieri, M Murdocca, G Novelli… - Human genomics, 2020 - Springer
Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system
represent two instruments of basic and translational research, which both allow to acquire …

DYRK1A and cognition: A lifelong relationship

ML Arbones, A Thomazeau… - Pharmacology & …, 2019 - Elsevier
The dosage of the serine threonine kinase DYRK1A is critical in the central nervous system
(CNS) during development and aging. This review analyzes the functions of this kinase by …