Human Stem Cell Use: iPS Cell
Human Cardiac Chip for Assessment of Proarrhythmic Risk
Research Objective This proposal will develop patient specific ‘heart-on-a-chip’ devices that will significantly impact early screening of drugs to accurately predict drug-induced proarrhythmia and toxicity. Impact Patient specific ‘heart-on-a-chip’ device will significantly reduce the cost of bringing a new drug candidate to market while improving efficacy. Major Proposed Activities To improve the maturity of human […]
A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs
Research Objective Develop a tool that facilitates rapid, cost effective development of optimized GMP-grade hPSC differentiation into functional DA neurons and apply this device to a cohort of PD patient-derived iPSCs. Impact Creating GMP-grade, functionally consistent phenotypes for DA neurons from each patient will significantly increase the likelihood of stem cell-derived DA neuron-based therapy for […]
A treatment for Zika virus infection and neuroprotection efficacy
Research Objective We propose to determine the impact of the Zika virus during human neurodevelopment and to test a FDA-approved therapeutic candidate to treat Zika infection. Impact A drug to treat/cure Zika infection and for neuroprotection. Major Proposed Activities To determine the molecular and cellular alterations caused by the Zika virus in the human developing […]
Targeted off-the-shelf immunotherapy to treat refractory cancers
Research Objective This project will use human pluripotent stem cells to produce a standardized, off-the-shelf immunotherapy using novel immune cells that are specifically targeted to cure otherwise lethal cancers. Impact Unlike current immunotherapies produced on a patient-specific basis, iPSC-derived immune cells are targeted to tumors with high specificity, no off-target effects and without need for […]
CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease
Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative process of Parkinson’s disease is a critical unmet medical need. Our approach is based on novel gene engineering technology that modifies expression of key target […]
Use of Human iPSC-derived Endothelial Cells for Calcific Aortic Valve Disease Therapeutics
Research Objective To develop drugs to treat Calcific Aortic Valve Disease (CAVD), the third leading cause of adult heart disease, by screening a stem cell-based platform based on CAVD patient-derived stem cells. Impact CAVD represents a major unmet medical need, with no treatments other than valve replacement. We will identify drugs, already proven to be […]
Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons
Research Objective Autologous human dopaminergic neurons derived from patient-specific induced pluripotent stem cells Impact Parkinson's disease Major Proposed Activities Characterize differentiation from all 10 patient cell lines Characterize functionality of patient neurons matured in vitro Immunogenicity assessment Cryopreservation feasibility testing Investigate dose response in vivo Detect dopamine release in vivo Thousands of Californians suffer from […]
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. […]
Novel Rejuvenated T Cell Immunotherapy for Lung Cancer
Research Objective Through this project, we would like to evaluate how this T-iPSC-based immunotherapy that we have developed can eliminate lung cancer cells effectively in vivo using xenografted SCID mice. Impact This novel T-iPSC-based immunotherapy will provide another effective treatment for lung cancer and possible other malignancies by supplying unlimited number of young and active […]
CRISPR/Cas9 nanoparticle enabled therapy for Duchenne Muscular Dystrophy in muscle stem cells
Research Objective Gene correction of muscle stem cells Impact These studies will develop a gene editing based therapy for one of the most prevalent lethal childhood disorders called Duchenne Muscular Dystrophy. Major Proposed Activities To identify the best MSNP-CRISPR candidates for CRISPR/Cas9 plasmid delivery in vitro to muscle stem cells To identify the best MSNP-STEM […]