Stage of Program: Basic or Applied Basic Research (DISC1 and many 1.0 Projects)


Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertension

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 and disease processes. Impact The studies aim to identify genetic targets for PAH therapy and develop a predictive model to accelerate stem cell research and […]

Lewy body dementia, 𝛼-synuclein, and cell-specific mechanisms of neurodegeneration

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 If the proposed studies are successfully achieved, they will impact our understanding of Lewy body dementias specifically and 𝛼-synucleinopathies and neurodegeneration more broadly. Major Proposed […]

Immune cloaking of human stem cell-derived insulin-producing cells for curative cell therapy without immunosuppression

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 escape rejection. Impact We test a novel molecule that reduces immune activation upon transplantation of allogeneic stem cell products that can improve graft survival and […]

Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells

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 single nuclei resolution. Impact These studies will elucidate satellite stem cell and myofiber defects in Duchenne and Becker Muscular Dystrophy and determine efficacy, mechanism and […]

Neuroimmune interactions in the developing human brain

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. Impact Neurodevelopmental disorders caused by immune cell perturbations will be impacted by the fundamental understanding of how immune system controls normal development of the brain. […]

Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell function

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 long-standing knowledge gaps related to the mechanisms of pro-regenerative impact of pregnant milieu and female muscle stem cell senescence Major Proposed Activities Isolation of female […]

Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cells

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 impact of our studies is the understanding of mechnistic differences between normal and cancer stem cells and the development of new therapies. Major Proposed Activities […]

Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment

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 neurodevelopmental disorders (NDDs), our work will identify and quantify cellular and molecular changes in human and mouse models of corticogenesis with NuRD deficiency. Major Proposed […]

Village-based identification of human risk factors for viral neuropathogenesis

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 translational discoveries. Impact Our work will identify the biological factors that influence inter-individual differences in susceptibility and immune response to neurotropic viruses, which could inform […]

Utilizing Age-Specific Adipocyte Progenitor Cells for Cell Therapy in Older Patients

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 hiPSCs. Impact Bottlenecks: 1) Older patients suffer from sarcopenic obesity, which has no safe and effective treatment. 2) Cell therapy in older patients is often […]

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