Research Objective We will identify SAR-CoV-2 T cell epitopes for vaccine development and specific TCRs for adoptive T cell therapy using a stem cell-based platform to generate specialized dendritic cells…
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 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 This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
Research Objective This DISC2 tests a strategy to target survival and self-renewal in glioma stem cells (GSC), and will identify a therapeutic candidate for safety, dosing and efficacy testing. Impact…
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 With a single administration to an individual, lentiviral vectors that selectively transduce B cells in vivo and express highly potent anti-HIV-1 proteins to suppress HIV-1 replication throughout life…
Research Objective This personalized chimeric antigen receptor (CAR) T cell therapy will attack a wider range of cancer cells, and block cancer's defense mechanisms to empower the immune system to…
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…
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