Human Stem Cell Use: Adult or Tissue Stem Cell
GENE EDITING FOR FOXP3 IN HUMAN HSC
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 HSCT, the only available treatment, has very poor outcomes including GvHD and low immune reconstitution. Major Proposed Activities Demonstrate specificity of targeted insertion of FOXP3 […]
Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells
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 of an approach to provide long-lasting functional cure for HIV infection, obviating the need for anti-retroviral therapy. Major Proposed Activities Test if engineered hematopoietic stem/progenitor […]
MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Research Objective Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease. Impact It could lead directly to a treatment for Angelman Syndrome, but the approach could be used to alter gene expression in almost any brain disorder. It could overcome the brain […]
Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration
Research Objective To develop a novel regenerative and adhesive hydrogel encapsulating patient's' gingival stem cell which can potentially be used as an adhesive dental hydrogel for periodontal tissue regeneration. Impact Upon successful completion, this project will introduce a promising treatment approach for maxillofacial defects presenting an innovative treatment modality for periodontal tissue regeneration. Major Proposed […]
Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α-hydroxylase for Acute Myeloid Leukemia Therapy
Research Objective We propose a new approach to differentiation therapy for acute myeloid leukemia by producing local level of high-dose vitamin D in bone marrow via cell therapy with engineered hematopoietic stem cells Impact If proven successful, the proposed research can serve as a major breakthrough in the treatment of multiple subtypes of AML and […]
Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion
Research Objective We aim to develop conditions for stable expansion of blood stem cells outside of the body Impact Blood stem cells are a rare but necessary cell type for curative bone marrow transplantation and related gene therapies. Stable blood stem cell expansion will increase therapy availability and success Major Proposed Activities Validate a fully […]
Development of treatments to improve healing of ischemic wounds
Research Objective We aim to develop an angiogenic proteoglycan mimic that will protect tissue from rapid degradation, and in conjunction with EPCs promote angiogenesis in order to accelerate ischemic wound healing. Impact As a treatment, LXW7-DS-SILY combined with a collagen scaffold and EPCs will accelerate healing of ischemic diabetic foot ulcers and reduce limb amputation […]
Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET
Research Objective This project aims to develop a sensitive and non-invasive method for tracking stem cells in clinical trial, without the need for genetically engineered reporters or long-lived radioisotopes. Impact The ability to see follow stem cells over time, as they engraft, will make it possible to predict response to stem cell therapy and understand […]
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 […]