Human Stem Cell Use: Adult or Tissue Stem Cell


Reprogramming human stem cells for blood cell generation

Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create a safe, unrestricted source of universal donor human blood cells that could be used to improve healthcare and save lives throughout the world. Major Proposed […]

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 […]

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 […]

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 […]

Targeting progenitors in scar tissue to reduce chronic scar burden

Research Objective Develop novel strategies to treat heart scars by targeting progenitors that replenish scars Impact There currently is no therapy for treating scar tissue in the heart or any other organ. Our proposal would lead to the development of targeted approaches to reduce scar burden. Major Proposed Activities Identify progenitors in scar tissue (murine […]

Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells

Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how acquired MPN-associated mutations cause disease by overproduction of various cell types and pave the way for gene editing therapies to reverse MPNs. Major Proposed Activities […]

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 […]

Exosomal Y-RNAs as mediators of bioactivity of cardiac-derived cell therapy

Research Objective We propose to dissect the contribution of Y-RNAs, small non-coding RNA species enriched in CDC-exosomes, in mediating the effect of CDC-exosomes on cardioprotection and macrophage polarization. Impact Examining the contribution of highly represented RNA species in CDC-exo could allow a better understanding of the mechanism of action of CDC-exo and modulation of their […]

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 […]