Platform Technology for Pluripotent Stem Cell-Derived T cell Immunotherapy
Research Objective We will combine a novel method to produce T cells from stem cells with gene editing tools, to create pluripotent stem cells that can serve as a universal…
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…
Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome
Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring…
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…
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…
A Novel Approach to Eradicate Cancer Stem Cells
Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and…
A Novel Approach to Eradicate Cancer Stem Cells
Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and…
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…
Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis
Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver…
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…
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