Human Stem Cell Use: iPS Cell
Drug Discovery for Dilated Cardiomyopathy using Patient-Derived Human iPSCs
Research Objective Greenstone Biosciences will use patient-derived stem cells to discover safe and effective drugs for cardiovascular disease. Impact The use of patient-derived stem cells to discover novel drug targets for cardiovascular disease, identify drug candidates, and establish clinical trial in a dish to evaluate drug safety and efficacy. Major Proposed Activities Use patient-derived induced […]
Developing a microglia replacement therapy
Research Objective To develop a new cell therapy for the brain by transplanting the brain's immune cells Impact The brain cell therapy we envision could be used to treat rare genetic metabolic diseases, Multiple Sclerosis, brain tumors, as well as common neurodegenerative diseases including Alzheimer's disease. Major Proposed Activities Finding the best source of human […]
Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine
Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the genetics of the patient and their cancer. Impact Success would provide proof-of-principle of a novel cell based therapy for cancers broadly. It is expected to […]
Development of a stem-cell based approach to interpret global effects of genetic variants contributing to neurodevelopmental disease risk
Research Objective We are developing a strategy to characterize the disease-relevance of hundreds of mutations across diverse genetic backgrounds using stem cells Impact Understanding how mutations impact cellular function can identify treatments for genetic diseases, but currently less than 1% of identified mutations have a known function. Major Proposed Activities Identify optimal conditions for SNV […]
Control of OCT4 abundance and function in human stem cells
Research Objective Our work will reveal an essential pathway that establishes precise levels of the OCT4 transcription factor and protects stem cell identity despite genetic or environmental stress. Impact Our work will enhance our ability to generate and expand high-quality induced pluripotent stem cells from older patients or patients of neurodegenerative disease for use in […]
Engineering AAV capsids for transduction of neural and muscle stem cells
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and brain. Impact New AAV capsids that target muscle and neural stem cells will enhance the number of neurological diseases able to be treated with AAV-based […]
Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells
Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve the siRNA delivery problem and rapidly open the door to Anti-COVID siRNA therapeutics. Major Proposed Activities Complete synthesis of a new delivery device called a […]
Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy
Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients with COVID-19. Impact The impact will be the avoidance of an animal model once an approved medication hit has been verified by our model. The […]
Chimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2
Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will provide a novel therapeutic approach to boost the cellular immunity against SARS-cov2, especially for high risk populations. Major Proposed Activities Construction of SARS-cov2 CAR constructs […]
Development of a novel PIKFYVE kinase inhibitor for the treatment of COVID-19
Research Objective Alveolar type II cells are the stem cells of the lung, and they are killed by SARS-CoV-2. We will determine if a novel PIKFYVE kinase inhibitor prevents SARS-CoV-2 infection of type II cells. Impact Although PIKFYVE inhibition blocks SARS-CoV-2 infection, no known PIKFYVE inhibitors have suitable drug-like properties. We would validate a novel […]