Human Stem Cell Use: Adult or Tissue Stem Cell
Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis
Research Objective Locally delivered formulation of RSpo1 protein as an activator of Lgr5+ epithelial stem cells in chemotherapy- or radiation therapy-induced oral mucositis Impact Oral mucositis Major Proposed Activities RSpo1 formulation design and selection for optimal oral delivery Activation of Wnt pathway by formulated RSpo1 in-vitro Production of RSpo1 protein Oral stem cell expansion by […]
Development of a novel stem-cell based carrier for intravenous delivery of oncolytic viruses
Research Objective Develop a stem cell-based platform that safely and efficiently delivers viruses that specifically kill tumor cells and restore immune activity in patients with advanced cancer. Impact Overcome the inherent limitations that prevent efficient intravenous delivery of tumor killing viruses to metastatic tumors. Major Proposed Activities Compare the relative efficacy of native or cargocyte-delivered […]
Preclinical development of an exhaustion-resistant CAR-T stem cell for cancer immunotherapy
Research Objective The expected outcome is an exhaustion-resistant CAR-T cell, which persists long-term in a functional progenitor T cell state in the tumor microenvironment and can be used for cancer immunotherapy. Impact CAR-T cells are effective in B cell cancer, but less than 50% of patients experience long-term disease control. Exhaustion-resistant CARs may provide long-term […]
Matrix Assisted Cell Transplantation of Promyogenic Fibroadipogenic Progenitor (FAP) Stem Cells
Research Objective We seek to develop a cell based-hydrogel therapy to improve outcomes in patients with muscle degeneration. The technology will improve muscle through sustained release of cell-based cytokines. Impact While designed for rotator cuff injuries based on the model, low back pain and spinal degeneration as well as traumatic muscle loss would be well […]
A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting
Research Objective Articular paste graft containing MSCs and an adhesive hydrogel that support cartilage growth will be combined for an effective and functional stem cell based cartilage repair procedure. Impact The proposed biologic cartilage repair therapy results in accessibility of an effective, low cost, one-step and functional biologic solution to those with cartilage injuries and […]
New noncoding RNA chemical entity for heart failure with preserved ejection fraction.
Research Objective Modified synthetic noncoding RNA molecule Impact Heart failure with preserved ejection fraction Major Proposed Activities Lead optimization Perform extensive preclinical testing and select a therapeutic candidate. Develop and test preliminary potency assays based on mechanistic insights. Demonstration of injury-modifying bioactivity in a clinically-relevant human progenitor cell population. Optimize formulation and dosing for intravenous […]
Development of a new therapeutic for directing target specific stem cell migration and treatment
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells Impact Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS Major Proposed Activities Complete […]
Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan Anemia (DBA). Impact If small molecule NLK inhibitors are identified that are effective in improving the anemia of DBA and nontoxic, then treatment and transfusions […]
The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease
Research Objective The goal of this project is to develop the first ORAL cell therapy as a breakthrough treatment for inflammatory bowel disease [IBD]. Impact We engineered a new way to deliver cells ORALLY instead of by injection. In doing so, we will better reach the inflamed tissues and provide a much-needed new treatment for […]
Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia
Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a relevant mouse model. Impact XLA can be treated with chronic immunoglobulin replacement, but may be sub-optimal due to infections and inflammatory complications. Stem cell gene […]