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.…
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 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 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 Elucidation of pregnancy-related factors that mitigate cellular senescence and enhance regeneration has far-reaching implications for understanding the mechanisms of aging and rejuvenation. Impact The study will address the…
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…
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…
Research Objective We will identify the risk factors underlying viral infections of the fetal brain using a novel human stem cell-based platform that has the potential to accelerate basic and…
Research Objective A new type of APC serves older patients as 1) better MSC in immunomodulation (reducing inflammation) for autologous transplantation; 2) better source of somatic cells for generating heathier…
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