Developing a microglia replacement therapy
Research Objective To develop a new cell therapy for the brain by transplanting the brain's immune cells Impact The brain cell therapy we envision could be used to treat rare…
Development of a stem-cell based approach to interpret global effects of genetic variants contributing to neurodevelopmental disease risk
Research Objective We are developing a strategy to characterize the disease-relevance of hundreds of mutations across diverse genetic backgrounds using stem cells Impact Understanding how mutations impact cellular function can…
Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine
Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the…
Control of OCT4 abundance and function in human stem cells
Research Objective Our work will reveal an essential pathway that establishes precise levels of the OCT4 transcription factor and protects stem cell identity despite genetic or environmental stress. Impact Our…
Engineering AAV capsids for transduction of neural and muscle stem cells
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and…
Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells
Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve…
Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy
Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients…
Chimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2
Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will…
Development of a novel PIKFYVE kinase inhibitor for the treatment of COVID-19
Research Objective Alveolar type II cells are the stem cells of the lung, and they are killed by SARS-CoV-2. We will determine if a novel PIKFYVE kinase inhibitor prevents SARS-CoV-2…
Repurposing Aminoadamantane Nitrate Compounds to Inhibit SARS-CoV-2 Viral Activity and to Protect the Brain from Viral-Related Damage
Research Objective The objective is to screen a series of aminoadamantane nitrate compounds for their ability to protect hiPSC-derived neurons from SARS-CoV-2-related damage and to block SARS-CoV-2 activity. Impact If…
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