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…
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…
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…
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…
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…
Research Objective We will build a foundational model and experimental platform to catalog all genes that promote and protect against PAH, with the potential to extend to many other developmental…
Research Objective We will describe, for the first time, human muscle satellite cell, myofiber and immune cell dynamics due to dystrophin deficiency and AAV gene therapy in human muscle at…
Research Objective This project will identify mechanisms by which immune cells regulate human brain development, and use stem cell derived models to identify pathological changes induced by congenital virus infection.…
Research Objective The goal of our project is to generate cells for replacement therapy in patients that have reduced ability to trigger the immune response in the recipient and therefore…
Research Objective We will generate insights about mechanisms by which 𝛼-synuclein leads to neurodegeneration of the forebrain and substantia nigra, regions affected in Lewy body dementias and Parkinson disease. Impact…
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