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…
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…
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…
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…
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…
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be…
Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
Research Objective Allogeneic human induced pluripotent stem cell-derived glial enriched progenitor cell therapy to treat mild traumatic brain injury Impact Prior to this study, glial cell-based therapies have never been…
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