Human Stem Cell Use: Embryonic Stem Cell
Assessing the Functional, Immunologic and Microbiologic Characteristics of Human Livers Created in Chimeric Pigs
Research Objective Generating human liver using pig as a bioreactor Impact The livers produced will be used for transplantation into patients with end-stage liver disease, metabolic disorders, and for metastatic liver disease not amenable to resection Major Proposed Activities MILESTONE 1 TASKS 1) Procure liver from control pig 2) Place on XVIVO Perfusion Device MILESTONE […]
Excitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injury
Research Objective The primary objective of this research is to test whether excitatory human V2a spinal interneurons engineered from PSCs can repair the damaged spinal cord and restore motor function. Impact Currently no existing therapies are capable of repairing the injured spinal cord. Our therapeutic cell candidate – human ‘V2a’ spinal interneurons – could address […]
Pluripotent Stem Cells for Tendon Tissue Engineering
Research Objective We propose to develop a bio-tendon engineered from differentiated pluripotent stem cells for the repair of tendon injuries and degeneration. Impact Rotator cuff tears are the most common causes of shoulder pain that require surgery. However, failure rates range from 20% to 90%. A successful tendon repair will have a major impact on […]
A Novel Therapy for Sanfilippo B
Research Objective To develop a Stem Cell therapy for Sanfilippo B syndrome. Impact There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal will develop a stem cell based therapy for Sanfilippo syndrome. Major Proposed Activities Generation of universal donor Embryonic Stem Cells (ESC H1) using state of […]
Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)
Research Objective Functional restoration following spinal cord injury using defined excitatory and inhibitory spinal interneuron progenitor cell transplantation Impact The development of a stem cell derived progenitor cell therapy with disease modifying potential for spinal cord injury Major Proposed Activities Define and characterize DI4 GABA and V3 Glut spinal interneuron progenitor cells derived from pluripotent […]
Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients
Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1 diabetes. Impact Our product will replace donor islets for cell replacement therapy in patients with type 1 diabetes, and will provide a readout of cell […]
Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models
Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini human retinas. Impact The research will use stem cell-based methods to overcome the shortage of human retinal cells and establish disease models, thus allow testing […]
Extending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1D
Research Objective Determine optimal islet transplant conditions and systemic treatments that promote graft survival upon transplantation into immune-competent diabetic subjects. Impact Our proposal will optimize the generation and viability of an unlimited, reproducible source of human engineered islets for transplantation. Major Proposed Activities Demonstrate improved HILO graft survival with FGF1 coating Prolong grafted HILO survival […]
iPSC-derived smooth muscle cell progenitor conditioned medium for treatment of pelvic organ prolapse
Research Objective Conditioned media from human iPSC-derived smooth muscle cell progenitors. This media exerts paracrine effect to restore damaged vaginal wall in patients with pelvic organ prolapse. Impact Pelvic organ prolapse (POP) is characterized by the downward movement of the vagina and/or uterus through the vaginal opening. It is treated with surgery. The candidate is […]
iPSC Extracellular Vesicles for Diabetes Therapy
Research Objective We will derive extracellular vesicles (EVs) from induced pluripotent stem cells (iPSCs), characterize the content and immunomodulatory activity of EVs, and deliver iPSC-EVs to treat Type-1 diabetes. Impact Type 1 diabetes (T1D) is an autoimmune disease and there is no therapy to preserve islet cells. Accomplishment of this project will generate a new […]