Research Objective Modified synthetic noncoding RNA molecule Impact Heart failure with preserved ejection fraction Major Proposed Activities Lead optimizationPerform extensive preclinical testing and select a therapeutic candidate.Develop and test preliminary…
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect…
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan…
Research Objective The goal of this project is to develop the first ORAL cell therapy as a breakthrough treatment for inflammatory bowel disease [IBD]. Impact We engineered a new way…
Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a…
Research Objective We aim to identify drug regimens that stimulate endogenous progenitors in the inner to regenerate to restore hearing or balance functions. Impact Treatment for irreversible hearing loss and…
Research Objective We propose to develop mesenchymal stem cell derived extracellular vesicles (MSC-EV) as treatment for lung fibrosis Impact MSC-EV are promising for several lung diseases, but we need to…
Research Objective The expected outcome is a therapeutic candidate, allogeneic HSC-engineered HLA-I/II-negative human iNKT cells, that can potentially be used as an off-the-shelf cellular therapy for treating cancer. Impact The…
Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As the…
Research Objective We are developing a tumor-associated glycan-targeting CAR T cell with inducible cytokine production that drives T cell stem/memory phenotype and persistence for effective treatment of ovarian cancer. Impact…
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