Therapeutic/Technology: Donor cell therapy
Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury.
Research Objective This project generates new cGMP compliant tissue educated human neural stem cell lines, paired with in vivo pre-clinical proof of concept testing, and development of a predictive in vitro profile. Impact Identification of new cell lines with in vivo efficacy testing to enable efficient translation to chronic cervical spinal cord injury, an area […]
iPS Glial Therapy for White Matter Stroke and Vascular Dementia
Research Objective This cell line will target recovery in ischemic white disease, a progressive dementing condition with no current therapy by developing a new stem line, iPS-glial enriched progenitors (iPS-GEPs). Impact This cell line will target tissue repair and recovery in ischemic white disease/vascular dementia, a chronically progressive and dementing condition with no current therapy. […]
Towards hepatocyte cell replacement therapy: developing a renewable source of human hepatocytes from pluripotent stem cells
Research Objective To develop a consistent and abundant source of transplantable human hepatocytes for transplantation. Impact Developing an abundant and consistent source of human hepatocytes that can be used to treat patients with liver failure. Major Proposed Activities To determine the degree by which human pluripotent stem cell (hPSC)-derived hepatocytes engraft and restore liver function […]
Neural Stem Cell Relays for Severe Spinal Cord Injury
Research Objective We propose to utilize human neural stem cells to form neuronal relays across sites of severe SCI, restoring function across the site of spinal cord injury. Impact We will develop a specific type of neural stem cell that is best suited for repairing the injured spinal cord. Major Proposed Activities In Vitro Assessment […]
Development of a Cellular Therapeutic for Treatment of Epilepsy
Research Objective A stem cell-derived nerve cell therapy to minimize seizures in people with epilepsy Impact Many people with epilepsy have uncontrolled seizures that can be life threatening and adversely impact quality of life and independence. A cell therapy could help those not responsive to drugs. Major Proposed Activities Transplant a nerve cell therapy made […]
Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0.
Research Objective We propose to discover the optimal human neural stem cell candidate for traumatic brain injury. 4 hNSC products (2 ES derived & 2 fetal) will be compared with TBI/vehicle controls, & then each other. Impact Traumatic brain injury (TBI) affects more Americans than brain, breast, colon, lung and prostate cancer combined ! There […]
Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI
Research Objective We developed technology to reproducibly prepare large numbers of bonafide cardiac progenitor cells from patient iPSCs. We propose the first test of these cells as a therapy for myocardial infarction. Impact Heart failure resulting from myocardial infarction is responsible for 13% of human mortality (WHO statistic). This proposed therapy is to restore the […]
Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells
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 progenitors alone. Impact The proposed studies would address key bottlenecks in cell replacement therapy for Type I Diabetes — issues with cellular engraftment, survival, and […]
Targeted off-the-shelf immunotherapy to treat refractory cancers
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 Unlike current immunotherapies produced on a patient-specific basis, iPSC-derived immune cells are targeted to tumors with high specificity, no off-target effects and without need for […]
Preclinical development of human hepatocyte progenitor cells for cell therapy
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 transplantation therapy can be an effective alternative treatment for severe liver diseases to liver transplantation, which is severely limited by the lack of available donor […]