Human Stem Cell Use: Embryonic Stem Cell
Meniscal Repair and Regeneration
Research Objective Stem cells are seeded into fibers spun out of collagen to fabricate tissue that resembles the knee meniscus Impact Meniscal tears are very common but do not heal. The treatment is removal of the torn tissue, which leads to osteoarthritis. If successful, replacing the tissue will prevent osteoarthritis. Major Proposed Activities Establish the […]
Hypoxia-specific Production of Exosomes from iPSC-derivatives for Myocardial Repair
Research Objective A lead therapeutic candidate will be selected: 1) exosomes from hypoxia-injured iPSC-derived cardiomyocytes (iCMs), 2) exosomal miRNA cluster, and 3) siRNA inhibition of exosomal target gene, Notch3. Impact Effective targeted therapy to restore the injured and vulnerable myocardium is urgently needed to reduce the high mortality of HF patients. Promising discovery of iPSC […]
Stem cell-derived extracellular vesicles to reverse radiation-induced brain injury
Research Objective These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition. Impact Stem cell-derived extracellular vesicles will address the confounders of stem cells (tumors, immunorejection, immunosuppression) & mitigate debilitating side-effects of cancer therapy on the brain. Major Proposed […]
Combating COVID-19 using human PSC-derived NK cells
Research Objective We propose to generate NK cells with enhanced immunity from gene-edited human PSCs and use the resultant NK cells to kill SARS-CoV-2-infected cells to combat against COVID-19. Impact The use of gene-edited hPSCs as a source for genetically engineered NK cells will allow us to generate effective immunotherapy for COVID-19 that has no […]
Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP)
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 impacted condition will be ALSP. However, further examination of the safety of human microglial transplantation will have broad implications for many neurodegenerative disorders Major Proposed […]
Dual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetes
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 and characterization of the injectable immunomodulating and pro-angiogenic material components: HA hydrogel, heparin nanoparticles and VEGF clusters. Generation of human islet like organoids from pluripotent […]
Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes
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 islets for transplantation into Type I diabetics. Major Proposed Activities Demonstrate efficacy of immune tolerant HILOs in humanized diabetic mice Demonstrate safety of immune tolerant […]
Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome
Research Objective We propose a platform to generate transplantable thymus organoids derived from human pluripotent stem cells designed to treat severe immunodeficiencies in children affected by 22q11 DS Impact Our product could impact 22q11DS and many other pathologies characterized by absence, degeneration or injury of the thymus and resulting in severe immunodeficiencies. Major Proposed Activities […]
Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer
Research Objective We will 1) identify non-invasive bladder cancer patients with (pre)cancerous urothelium by single-cell RNA-seq and 2) replace this dangerous lesion with normal hESC-derived bladder progenitors. Impact Replacement of corrupted (pre)cancerous urothelium with pluripotent cell-derived normal bladder progenitors will provide a definitive treatment for bladder cancer, expected to eliminate recurrence. Major Proposed Activities To […]
Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatment
Research Objective We propose to develop a cell encapsulation technology to support the long term viability and function of human stem cell derived insulin producing cells. Impact A device that provides adequate mass transfer of oxygen, glucose, and insulin for encapsulated stem cell derived beta cells can address the challenges of current cell therapy for […]