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 This project investigates how normal tau protein protects aging neurons. We'll use aging human neurons to understand how tau helps repairing DNA and why this protection fails disease.…
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…
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.…
Research Objective We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less…
Research Objective We will identify genetic regulators and intercellular signals that promote islet cell function, and apply our work to advance stem cell biology for generating replacement islets to reverse…
Research Objective We will determine how viral infections alter lung progenitor cells thereby impairing their ability to regenerate the lungs and in turn promoting lung fibrosis. Impact We will define…
Research Objective We are building a foundational platform for developing genome editing technologies that increase patient coverage by 20-40X and can be applied to over 700 genetic diseases in diverse…
Research Objective We aim to solve a 30 year old problem, which is to understand using human stem cells how certain genetic mutations cause human disease. Impact We currently don't…
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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