Research Objective Development of hESC-derived pancreatic beta cells that are protected from allogeneic and autoimmune attack into a cell therapy for type 1 diabetes (T1D) Impact Cell therapy of T1D…
Research Objective Therapeutic candidate to cure hemophilia A is AAV-based genome editing vector that corrects the disease-causing mutation in the factor VIII gene in patient stem cells to develop a…
Research Objective A stem cell-derived nerve cell therapy to minimize seizures in people with epilepsy Impact Many people with epilepsy have uncontrolled seizures that can be life threatening and adversely…
Research Objective We propose to discover the optimal human neural stem cell candidate for traumatic brain injury. 4 hNSC products (2 ES derived & 2 fetal) will be compared with…
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…
Research Objective The objective of the proposed research is to perform 3 independent hESC-based screens to identify drug candidates for Huntington’s Disease. Impact There are currently no effective treatments for…
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…
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…
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…
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…
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