Research Objective We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will…
Research Objective We propose to investigate the transplantation of pluripotent stem cell derived microglia as a potential therapy for the devastating neurological disease; Adult-onset leukoencephalopathy (ALSP/HDLS). Impact The most immediately…
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 Functional human islet like organoids differentiated from human pluripotent stem cells. Impact Providing the immediate cell therapeutic candidate for clinical trial of diabetic patients. Major Proposed Activities Fabrication…
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 These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in…
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 Develop stem cell-based therapy to treat dementia Impact There are no treatments for dementia. If successfully achieved, this study will lead to a cure of a familial form…
Research Objective Development of immune tolerant human islet-like organoids for transplantation into diabetic patients. Impact Our proposal will progress the development of an unlimited, reproducible source of immune tolerant engineered…
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