Program Type: Discovery
Prodrug innovation to target muscle stem cells and enhance muscle regeneration
Research Objective To target therapeutics to muscle stem cells, the building blocks of skeletal muscle. Impact Drugs, genes and gene editing strategies can be delivered directly to muscle stem cells to alleviate disease. Major Proposed Activities Synthetic peptides based on the ectodomains of Myomaker will be synthesized, with a fluorophore conjugated for tracking. Alternatively, anti-Myomaker […]
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 […]
Hypo-immunogenic cardiac patches for myocardial regeneration
Research Objective To engineer a cardiac patch to restore function after a heart attack while avoiding an immune response (“hypo-immunogeneic” CP) when transplanted into a genetically distinct (“allogenic”) individual. Impact By making hypo-immunogeneic CPs and functional cardiac cells (induced pluripotent stem, “iPS” cells) available to commercial/research entities, our study can fuel the transformation of healthcare. […]
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 […]
Reverse transcriptase inhibitors as a novel therapeutic approach for neurological autoimmune disorders
Research Objective We found that approved anti-retroviral drugs could stop inflammation and block neurodegeneration. We propose to validate the re-purpose efficacy of these clinically-approved retroviral drugs. Impact We have identified an unexpected cause to a brain inflammation and a potential simple treatment. Our research could help millions of patients affected by a broad range neuro-immunological […]
Novel Platforms to Enhance In Vivo Delivery of Skeletal Muscle Progenitor Cells from Human Pluripotent Stem Cells
Research Objective Delivery of muscle stem cells presents a major roadblock for therapy. We explore novel approaches to increase the efficiency of delivering and monitoring muscle stem cells derived from hPSCs. Impact Development of enhanced monitoring and delivery platforms will greatly accelerate translational strategies aimed at delivering muscle stem cells for transplantation to patients with […]