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 The research will generate the know-how for replacing lost or injured bile ducts in the liver by reprogramming an abundant and related cell type that normally has a…
Research Objective Develop a high-throughput platform combining iPSC-derived cardiac organoids and CRISPR base editing to functionally assess missense variants in hypertrophic cardiomyopathy. Impact (i) Limited tools for functional interpretation of…
Research Objective 1.- Develop a better chronic pain in vitro model using iPSC-derived nociceptors. 2.- Understand the Mechanism of Action behind Chronic Pain Nociception. 3.- Attributes to establish a robust…
Research Objective This research will define the dynamic mechanisms of human embryo implantation by integrating live imaging, spatial multi-omics, and gene editing in physiologically relevant stem cell-based models. Impact This…
Research Objective Development of a human stem cell-derived, spatially-patterned, mature and functional human synthetic kidney organoid (hSKO) model as a platform technology for basic and translational kidney research. Impact Lack…
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…
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…
Research Objective We will develop genome editing tools to identify silencers that regulate neural stem cell fate, uncovering key DNA elements that guide neurodevelopment and are disrupted in neurodevelopmental diseases…
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…
