Human Stem Cell Use: Adult or Tissue Stem Cell

Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas

Research Objective We will develop a non-viral gene editing technology to replace the endogenous TCRα locus of stem memory T cells with transgene TCRs that are specific to brain cancer neoantigens. Impact Gliomas are lethal tumors often affecting children and young adults. Therapy using Tscm directed to attack truncal neoantigens in these tumors may provide […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 stem cell gene therapy for X-HIGM closer to the clinic, as there are currently no options for those without an HLA match or with infections […]

Genome Editing to Correct Cystic Fibrosis Mutations in Airway Stem Cells

Research Objective Gene corrected autologous airway epithelial stem cells from patients with cystic fibrosis to be used as cell and gene based therapy for chronic sinus disease Impact The proposed studies would provide an innovative, readily applied primary stem cell based approach with gene correction to treat chronic sinusitis in CF, a debilitating airway disease. […]

Protein tyrosine phosphatase – sigma inhibitors for hematopoietic regeneration

Research Objective We propose to develop a lead small molecule inhibitor of PTPσ, a receptor expressed by human blood stem cells, for the purpose of promoting human hematopoietic regeneration. Impact Systemic administration of a PTPσ inhibitor can accelerate hematologic recovery in thousands of patients who have received myelosuppressive chemo- or radiotherapy. Major Proposed Activities Generate […]

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 […]