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…
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…
Interrogation of tandem repeat variants contributing to neurodevelopmental and psychiatric traits using stem cell models
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…
A novel platform to rescue neurodevelopmental disorders caused by haploinsufficiency
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
Mechanisms underlying dosage sensitivity in developmental disorders
Research Objective This proposal leverages pluripotent stem cell models to understand the principles by which changes in gene and protein dosage affect human neural and facial progenitor development and cause…
Modeling Rett syndrome neurological disorder with human pluripotent stem cells to develop in cellulo screening platforms.
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.…
Ex vivo fate mapping of human lung stem cell plasticity in fibrotic disease
Research Objective This proposal will design new models to study behavior that is specific to human lung stem cells, and screen for drugs that can target abnormal stem cells in…
Understanding Chemotherapy-Induced Peripheral Neuropathy Mechanisms using CRISPRi and Chemical Screens in Human iPSC-Derived Sensory Neurons
Research Objective The research objectives are to identify causal genes for chemotherapy-induced mitochondrial toxicity and neurodegeneration in sensory neurons and drugs that target this toxicity. Impact These studies will open…
Stem cell-based rapid identification of SARS-CoV-2 T cell epitopes and T cell receptors for therapeutic use
Research Objective We will identify SAR-CoV-2 T cell epitopes for vaccine development and specific TCRs for adoptive T cell therapy using a stem cell-based platform to generate specialized dendritic cells…