Therapeutic/Technology: Therapeutic Approach
Curing bladder cancer by replacing corrupted urothelium with differentiated hES cells
Research Objective The goal of the proposed research is to use human embryonic stem cells to generate bladder epithelial progenitor cells that can be used to replace a cancerous bladder epithelium in vivo. Impact The long-term goal of the proposed research is to cure bladder cancer. Successful completion of this work may indicate that transplantation […]
Autologous iPSC-based therapy for radiation induced bladder injury
Research Objective To explore if iPSC-based therapy can prevent bladder damage due to radiation therapy, thereby limiting the unintended consequences of treatments for prostate, gynecologic and colorectal cancers. Impact This therapy impacts cancer survivors by preventing the permanent debilitating urinary symptoms due to radiation therapy. Currently there are no therapies to prevent radiation bladder damage. […]
iPS-Interneuron Transplantation for Neural Repair after Stroke
Research Objective To determine if transplantation of iPS-interneurons cells (iPS-3i cells) enhances functional recovery in stroke. Impact Successful completion of the proposed studies will develop a brain repair therapy for stroke, an unmet clinical need with significant impact on society. Major Proposed Activities To determine the recovery effect of transplantation of iPS-3i cells in the […]
Examining the efficacy of GDF11 antibody as a rejuvenator of aged human muscle stem cell capacity and muscle repair .
Research Objective To examine the efficacy of blocking blood borne GDF11 activity to rejuvenate aged human muscle stem cell regenerative capacity. Impact This project will provide a proof-of-principle that GDF11 inhibition can boost aged human skeletal muscle repair, and facilitate its translational potential. Major Proposed Activities Determine GDF11 protein levels in human sera as a […]
Activation of patient-specific endogenous myocardial repair through the exosomes generated from the hypoxic iPSC-derived cardiomyocytes (iCMs).
Research Objective This proposal will provide direct evidence of clinical implementation of patient-specific iPSC products by validating the efficacy of autologous, cell-free exosome therapy. Impact Five-year survival of heart failure is a dismal 50% and is top diagnosis of hospital admission. Exosomes offer a feasible and effective cell-free therapy by activating endogenous myocardial repair. Major […]
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]
Living Synthetic Vascular Grafts with Renewable Endothelium
Translational Candidate LXW7 coated ePTFE vascular graft achieves rapid endothelization and improved graft patency by capturing endogenous endothelial progenitor cells Area of Impact This technology will produce long-lasting vascular grafts with self-renewable “living” endothelium and improve dialysis patients’ quality of life Mechanism of Action The arteriovenous ePTFE dialysis graft approach is the most common form […]
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility Mechanism of Action The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a […]
Targeting pancreatic cancer with Allogeneic Off-the-Shelf PSCA-CAR NK cells
Translational Candidate PSCA-CAR_sIL15 NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with metastatic pancreatic cancer or other cancers that also highly express PSCA Mechanism of Action PSCA-CAR_sIL15 NK cells are umbilical cord blood-derived CD34+ HSCs that are engineered to target PSCA and express soluble IL-15, and then are […]
Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells
Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells undergo genome editing to restore the deficient enzyme. Reintroducing these edited cells replaces the patient's bone marrow, establishing a lasting enzyme reservoir. The bone marrow […]