Human Stem Cell Use: iPS Cell
The role of WNT and BMP signaling pathways in iPSC to iTenocyte step-wise differentiation for tendon repair
Research Objective Development-inspired differentiation will enable efficient and specific generation of tenocytes that can repair tendon injury, restore dysfunctional tissue, and prevent long term effects. Impact This study will eliminate heterogenous differentiation of pluripotent stem cells and will results in high yield and unified tenogenic phenotype. Major Proposed Activities Establish the mechanism by which Wnt […]
Developing a Human Model of Sporadic ALS Using Machine Learning and Robotic Microscopy
Research Objective We will develop the first human stem cell model of sporadic ALS (sALS) to identify disease mechanisms in the most common form of ALS and to discover drugs to treat the vast majority of ALS patients. Impact Failure of drugs to treat sALS may be due to the use of models of familial […]
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 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 […]
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 […]
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 […]