Program Type: Discovery
Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatment
Research Objective We propose to develop a cell encapsulation technology to support the long term viability and function of human stem cell derived insulin producing cells. Impact A device that provides adequate mass transfer of oxygen, glucose, and insulin for encapsulated stem cell derived beta cells can address the challenges of current cell therapy for […]
Engineering Lifelong Cellular Immunity to HIV
Research Objective We aim to uncover a therapeutic approach to attempt to treat and potentially cure HIV infection using gene modified blood forming stem cells to enhance the immune response against HIV. Impact The study will allow a potentially curative treatment for HIV infection, which currently doesn’t exist. This will eliminate the need to administer […]
Targeting Cancer Stem Cells in Hematologic Malignancies
Research Objective We will develop a biotherapeutic/monoclonal antibody that blocks the growth of human AML cancer stem cells in vitro and in vivo. Impact Treatment of the cancer stem cell driven disease Acute Myelogenous Leukemia (AML) will be impacted. AML is the most common acute leukemia in adults and current treatments are largely ineffective. Major […]
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. […]
Identification and Generation of Long Term Repopulating Human Muscle Stem Cells from Human Pluripotent Stem Cells
Research Objective We will molecularly and functionally define muscle stem cells in human muscle in development, juvenile and adult and develop strategies to generate the most regenerative muscle stem cells from hPSCs. Impact There is no clinically relevant cell endowed with continuous repopulation ability from hPSCs. This work could provide a cell therapy for muscle […]
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 […]
Stimulating endogenous muscle stem cells to counter muscle atrophy
Research Objective Intramuscular delivery of two repurposed FDA approved drugs will activate resident muscle stem cells. This therapeutic strategy will augment regeneration and restore strength to atrophied muscles. Impact Currently effective treatments are lacking for localized muscle atrophy due to nerve injury (eg., Carpal Tunnel Syndrome) or immobilization after trauma or surgery (eg., hip or […]
Preclinical development of an immune evasive islet cell replacement therapy for type 1 diabetes
Research Objective We will produce a universal donor cell (UDC) line by gene editing an embryonic stem cell line. Cell therapies produced from the UDC line will not be rejected by a patient’s immune system. Impact The UDC line will address the bottleneck of patient immunity that is currently slowing development of many potential cell […]
Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs
Research Objective Development of hESC-derived pancreatic beta cells that are protected from allogeneic and autoimmune attack into a cell therapy for type 1 diabetes (T1D) Impact Cell therapy of T1D is challenged by immune rejection. Therefore, we will develop pancreatic progenitors derived from genetically modified hESCs that can evade allogeneic and autoimmune responses. Major Proposed […]