Project Objective: Research Insights


Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy

Research Objective The goal of this proposal is to define protocols to generate expandable, self-renewing human muscle stem cells (MuSC) from hiPS cells for Duchenne Muscular Dystrophy disease modeling and therapeutics. Impact The integration of STAT3i with current approaches to derive myogenic cells from hiPS cells would enable the generation of self-renewing MuSC that are […]

A Novel Tissue Engineering Technique to Repair Degenerated Retina

Research Objective Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases Impact Based on the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal […]

Developing a personalized approach to beta cell replacement for patients with a genetic form of diabetes

Research Objective To correct a gene mutation in a patient's stem cells and produce functional replacement cells for the patient to cure their diabetes. Impact WE expect that this project can serve as a model for developing new treatments for patients with certain forms of genetic diabetes. Major Proposed Activities To understand how the patient's […]

Modulating Liver Sinusoidal Endothelial Cell Permeability to Enhance Engraftment of Endothelial Cell Progenitors for the Treatment of Hemophilia A

Research Objective We aim to demonstrate that regulators of endothelial cell permeability can foster engraftment of endothelial cell progenitors in the liver sinusoids leading to production of Factor VIII. Impact Our work would provide conceptual proof that a cell based therapy for hemophilia A is possible and should be pursued. Major Proposed Activities Demonstrate that […]

Embryonic Stem Cells for Corneal Endothelial Degeneration

Research Objective The proposed studies will determine the optimal approaches to differentiate and transplant stem cell-derived corneal endothelial cells. Impact These data will provide foundational proof-of-concept data that will allow the rapid advance of a cell therapy towards clinical application. Major Proposed Activities Determine optimal conditions to generate human corneal endothelial cells from human stem […]

Identification of stem cell surface markers as potential therapeutic targets for advanced prostate cancer

Research Objective The goal of this proposal is to identify proteins found on the surface of both human prostate stem cells and cancer cells that could be used as potential targets for treating advanced prostate cancer. Impact There is no cure for advanced prostate cancer. This combined with the success of treating other cancers by […]

Generation of bile duct-competent transplantable human liver organoids

Research Objective Generation of human stem cell-derived mini livers capable of exporting bile into the gallbladder after transplantation into the liver Impact Mini livers capable of normal bile export would have potential for therapy of diseases in which bile export is impaired like Alagille syndrome Major Proposed Activities Generation of mini livers using human stem […]

New Methods for the Chemical Expansion of Hematopoietic Stem and Progenitor Cells

Research Objective We will develop a new agent that can increase the production of hematopoietic stem and progenitor cells and determine how the compound functions Impact We aim to develop a method to achieve the highest fold expansion of hematopoietic stem cells from a single unit of cord blood achieved to date increasing the supply […]

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 […]