Multi-gene modulation to rescue CNS-associated microdeletion syndromes
Research Objective Chromosomal microdeletion syndromes result in severe neuropsychiatric syndromes and lack therapy. This proposal will define critical genomic regions needed to generate new tools for functional rescue. Impact This…
Modeling the genetic basis of psychopathology in schizophrenia and autism
Research Objective We propose to apply scalable platforms for the characterization of CNVs and genes coupled with detailed characterizations of neurodevelopment of the 4 major CNV loci in model systems…
Defining Neurovascular Metabolism in Neurodevelopmental and Neuropsychiatric Disorders
Research Objective Neuropsychiatric disorders correlate to impaired metabolism, though understudied. We will describe how metabolism impacts disorders etiology and identify new, tractable therapeutic strategies. Impact We will uncover metabolic drivers…
CIRM Center for Neuropsychiatric Stem Cell Proteomics
Research Objective This project will interrogate interactions, distribution, and function of high-confidence neuropsychiatric disorder risk proteins, and identify convergent pathobiology of patient genetic variants. Impact Datasets and stem cell resources…
Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments
Research Objective We will uncover pathways through which ASD mutations cause disease and close the gap from disease research to therapeutic testing using organoids, primary human neurons, machine learning and…
Multiomic Studies of Idiopathic Intellectual Disability and Autism Spectrum Disorder (ID/ASD)
Research Objective We provide mechanistic insight into idiopathic Intellectual Disability (ID) and Autism Spectrum Disorder (ASD) based on aberrant redox-mediated posttranslational modifications related to air pollution Impact How air pollution…
Translational epigenomics: dissecting cell type-specific function of neuropsychiatric risk genes in vivo
Research Objective Our objective is to enable scalable genetic screening to study how different cell types and epigenetic networks are impacted by risk genes implicated in human psychiatric disorders. Impact…
A Center for Stem Cell Disease Modeling and Therapeutics
The goal of this project is to support the discovery and evaluation of novel therapeutics using stem cell-based models and drug and CRISPR screening. The facility will provide California researchers…
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