Deep phenotyping of human brain organoid models of autism spectrum disorder to unravel disease heterogeneity and develop biomarkers and treatments

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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…

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Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models

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Research Objective Our project will identify molecular and cellular changes induced by specific genetic variants implicated in schizophrenia and autism spectrum disorder in stem cells, neuroprogenitor cells and neurons. Impact…

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In neurons and beyond: how protein interactions shape the cellular response to Huntington’s Disease

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Research Objective New knowledge relating to protein-protein interactions that drive vulnerability of different cell types to neurodegeneration disease mutations using Huntington's disease as a paradigm. Impact The studies will inform…

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Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.

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Research Objective Study the neurological disorder Rett syndrome using neurons derived from genetically engineered human pluripotent stem cells and develop platforms to screen drugs for this disease in live cells.…

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Immune cloaking of human stem cell-derived insulin-producing cells for curative cell therapy without immunosuppression

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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…

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Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment

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Research Objective Human and animal models of NuRD-deficiency will identify NuRD-subtype function in context of neurogenesis. Multi-omic studies will identify/quantify molecular and cellular changes in NuRD-deficiency. Impact NuRD-deficiency causes several…

Continue ReadingModeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment