Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a…
Research Objective These studies will determine whether stem cell derived exosomes (nano-scale vesicles) can be used to treat the symptoms of Alzheimer’s disease (AD). Impact Our stem cell-derived exosome therapy…
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create…
Research Objective To target therapeutics to muscle stem cells, the building blocks of skeletal muscle. Impact Drugs, genes and gene editing strategies can be delivered directly to muscle stem cells…
Research Objective The goal of this proposal is to define protocols to generate expandable, self-renewing human muscle stem cells (MuSC) from hiPS cells for Duchenne Muscular Dystrophy disease modeling and…
Research Objective To engineer a cardiac patch to restore function after a heart attack while avoiding an immune response (“hypo-immunogeneic” CP) when transplanted into a genetically distinct (“allogenic”) individual. Impact…
Research Objective Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases Impact Based on…
Research Objective To correct a gene mutation in a patient's stem cells and produce functional replacement cells for the patient to cure their diabetes. Impact WE expect that this project…
Research Objective We aim to demonstrate that regulators of endothelial cell permeability can foster engraftment of endothelial cell progenitors in the liver sinusoids leading to production of Factor VIII. Impact…
Research Objective The proposed studies will determine the optimal approaches to differentiate and transplant stem cell-derived corneal endothelial cells. Impact These data will provide foundational proof-of-concept data that will allow…
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