Research Objective The expected outcome of these studies is a cellular therapeutic for Type I Diabetes: engineered human islets for transplant into patients, surpassing the function of beta cells or…
Research Objective We propose to develop a lead small molecule inhibitor of PTPσ, a receptor expressed by human blood stem cells, for the purpose of promoting human hematopoietic regeneration. Impact…
Research Objective This project will use human pluripotent stem cells to produce a standardized, off-the-shelf immunotherapy using novel immune cells that are specifically targeted to cure otherwise lethal cancers. Impact…
Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative…
Research Objective To develop a gene therapy product to deliver cardiac reprogramming factors into the heart for regeneration of new heart muscle. Impact The proposed candidate would regenerate heart muscle…
Research Objective The therapeutic candidate is a human Neural Stem Cell that secretes a protein, ApiCCT1, that aids in the prevention of disease phenotypes, for application in treatment of Huntington's…
Research Objective Determine if human hepatocyte progenitor cells, which exist in the normal adult liver, can be maintained and expanded in vitro while maintaining in vivo regenerative capacity. Impact Cell…
Research Objective We propose to develop a macroencapsulation technology, based on flexible nanoporous thin films, to support the long term viability and function of human stem cell derived insulin producing…
Research Objective CRISPR/Cas9 mediated FOXP3 gene editing in patient-derived hematopoietic stem cells as a cure for IPEX syndrome Impact FOXP3 mutation in IPEX syndrome leads to immune system dysregulation. Allogeneic…
Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy…
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