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 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 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 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 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 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…
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 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 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 study will address gaps in our understanding of how normal and cancer stem cells differ in their epigenetic states, helping develop new cancer-stem-cell-targeting therapis. Impact The long-term…
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