Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)


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 […]

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 […]

Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction of Hemophilia A

Research Objective Therapeutic candidate to cure hemophilia A is AAV-based genome editing vector that corrects the disease-causing mutation in the factor VIII gene in patient stem cells to develop a permanent cure. Impact Permanent correction of hemophilia A by editing mutations in the FVIII gene in stem cells. Develop a precise and efficient non-nuclease genome […]

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 […]

Immunization strategies to prevent Zika viral congenital eye and brain disease

Research Objective Our objective is to utilize human iPSC-derived neural and ocular cells to identify growth attenuated and non-pathogenic Zika virus vaccine candidates that can prevent congenital ZIKV disease. Impact Currently, there are no therapies or vaccines available against ZIKV for human use. The human iPSC technology provides a unique opportunity to test the growth […]

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