Project Objective: Development Candidate
Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs
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 is challenged by immune rejection. Therefore, we will develop pancreatic progenitors derived from genetically modified hESCs that can evade allogeneic and autoimmune responses. Major Proposed […]
Development of a Cellular Therapeutic for Treatment of Epilepsy
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 impact quality of life and independence. A cell therapy could help those not responsive to drugs. Major Proposed Activities Transplant a nerve cell therapy made […]
Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction of Hemophilia A
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 permanent cure. Impact Permanent correction of hemophilia A by editing mutations in the FVIII gene in stem cells. Develop a precise and efficient non-nuclease genome […]
Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0.
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 TBI/vehicle controls, & then each other. Impact Traumatic brain injury (TBI) affects more Americans than brain, breast, colon, lung and prostate cancer combined ! There […]
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 […]
Discovery of therapeutics for Huntington’s Disease
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 HD. Combination of human isogenic HD-mutants, novel tools and technology will provide therapeutic solutions for this neurodegenerative orphan disease. Major Proposed Activities Screening of 2,000 […]
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 stem cell gene therapy for X-HIGM closer to the clinic, as there are currently no options for those without an HLA match or with infections […]
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 […]
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 self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]
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 self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]