Human Stem Cell Use: iPS Cell
Identification and Generation of Long Term Repopulating Human Muscle Stem Cells from Human Pluripotent Stem Cells
Research Objective We will molecularly and functionally define muscle stem cells in human muscle in development, juvenile and adult and develop strategies to generate the most regenerative muscle stem cells from hPSCs. Impact There is no clinically relevant cell endowed with continuous repopulation ability from hPSCs. This work could provide a cell therapy for muscle […]
Immunization strategies to prevent Zika viral congenital eye and brain disease
Research Objective Our objective is to utilize human iPSC-derived neural and ocular cells to identify growth attenuated and non-pathogenic Zika virus vaccine candidates that can prevent congenital ZIKV disease. Impact Currently, there are no therapies or vaccines available against ZIKV for human use. The human iPSC technology provides a unique opportunity to test the growth […]
Non-Toxic, Highly-Effective Bioinspired Cryoprotectants for On-Demand Stem Cell Therapies
Research Objective An advanced technology is sought to replace the toxic, ineffective and highly processed components in legacy cryopreservation media. This technology is a fundamentally novel non-toxic freezing media. Impact The transport and storage of stem cell therapies is crippled by freezing media with poor cell preservation. Significantly improved freezing media would directly increase therapy […]
Microenvironment for hiPSC-derived pacemaking cardiomyocytes
Research Objective This proposal investigates the effects of the microenvironment on the development and maintenance of pacemaking function in human induced pluripotent stem cell (hiPSC)-derived cardiomyocytes. Impact Pacemaking function of hiPSC-derived cardiomyocytes is lost over time. Sustainability of pacemaking function of these cells is critical for engineering an biopacemaker from the patient's own cells. Major […]
Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI
Research Objective We developed technology to reproducibly prepare large numbers of bonafide cardiac progenitor cells from patient iPSCs. We propose the first test of these cells as a therapy for myocardial infarction. Impact Heart failure resulting from myocardial infarction is responsible for 13% of human mortality (WHO statistic). This proposed therapy is to restore the […]
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 […]