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 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 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…
Research Objective Understanding alveolar progenitor cell defects in T21 and the genes/pathways associated with them will allow for developing therapeutic approaches for individuals with DS. Impact Although trisomy 21 affects…
Research Objective This study will use pluripotent stem cells derived from patients to determine why Intellectual Disabilities caused by mutations in chromatin regulatory proteins leads to neuronal defects. Impact Our…
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders.…
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