Human Stem Cell Use: Embryonic Stem Cell
Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]
Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy
Research Objective This study examine potential immunologic changes caused by cellular reprogramming that could present a barrier to clinical application of regenerative therapies. Impact Identification and evaluation of immunologic changes caused by cellular reprogramming provides critical information to maximize the efficacy and safety of regenerative cellualar therapies. Major Proposed Activities Identify changes to the repertoire […]
An exosome-based translational strategy to mitigate Alzheimer’s disease neuropathology
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 will provide a viable approach to ameliorate the relentless progression of AD that severely impacts quality of life for millions of patients and their families. […]
Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy
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 therapeutics. Impact The integration of STAT3i with current approaches to derive myogenic cells from hiPS cells would enable the generation of self-renewing MuSC that are […]
A Novel Tissue Engineering Technique to Repair Degenerated Retina
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 the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal […]
Embryonic Stem Cells for Corneal Endothelial Degeneration
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 the rapid advance of a cell therapy towards clinical application. Major Proposed Activities Determine optimal conditions to generate human corneal endothelial cells from human stem […]
Reverse transcriptase inhibitors as a novel therapeutic approach for neurological autoimmune disorders
Research Objective We found that approved anti-retroviral drugs could stop inflammation and block neurodegeneration. We propose to validate the re-purpose efficacy of these clinically-approved retroviral drugs. Impact We have identified an unexpected cause to a brain inflammation and a potential simple treatment. Our research could help millions of patients affected by a broad range neuro-immunological […]
Organoid Modeling of Human Cortical Microcircuits
Research Objective The proposed studies will develop three-dimensional cell culture methods for creating human brain neural circuits for disease research and drug discovery. Impact The proposed research will develop a new research platform for studying how neurons in the human brain function, how neurological disease subverts this activity, and how we might find new therapies. […]
Curing bladder cancer by replacing corrupted urothelium with differentiated hES cells
Research Objective The goal of the proposed research is to use human embryonic stem cells to generate bladder epithelial progenitor cells that can be used to replace a cancerous bladder epithelium in vivo. Impact The long-term goal of the proposed research is to cure bladder cancer. Successful completion of this work may indicate that transplantation […]
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility Mechanism of Action The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a […]