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…
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…
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…
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…
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…
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…
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…
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…
Discovery of therapeutics for Huntington’s Disease
Research Objective The objective of the proposed research is to perform 3 independent hESC-based screens to identify drug candidates for Huntington’s Disease. Impact There are currently no effective treatments for…
Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome
Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring…
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