Cell Line Generation: Adult or Tissue Stem Cell
Harnessing vascular stem cells to grow and protect the human brain
Research Objective The origins of brain vascular mural cells are unknown. This proposal will identify mural stem cells in the developing human and mouse brain and determine their impact on blood brain barrier formation. Impact Brain vascular diseases can have profound impacts on long-term neurological function. This proposal will map the stages of mural stem […]
Determining how age-specific heterogeneity of human hematopoietic stem cells and megakaryocyte progenitors contribute to thrombotic disease upon aging
Research Objective Our research will determine how aging of human blood stem cells leads to dramatic increases in disorders of platelets, cells that normally prevent bleeding but form harmful clots when dysregulated. Impact Our findings have the potential to inform prevention and mitigation strategies of bleeding and clotting disorders that contribute to significant morbidity and […]
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]
A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting
Research Objective Articular paste graft containing MSCs and an adhesive hydrogel that support cartilage growth will be combined for an effective and functional stem cell based cartilage repair procedure. Impact The proposed biologic cartilage repair therapy results in accessibility of an effective, low cost, one-step and functional biologic solution to those with cartilage injuries and […]
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 […]
Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells
Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy of an approach to provide long-lasting functional cure for HIV infection, obviating the need for anti-retroviral therapy. Major Proposed Activities Test if engineered hematopoietic stem/progenitor […]
Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)
Translational Candidate Autologous CD34+ hematopoietic stem and progenitor cells (HSPC) with BTK gene insertion for treatment of X-linked agammaglobulinemia (XLA). Area of Impact The candidate will provide improved outcomes for patients with XLA, by allowing autologous transplantation with reduced intensity conditioning Mechanism of Action The Drug Product has biological activity of hematopoietic stem cells (HSC) […]
Extracellular Vesicle-Based Therapy for Corneal Scars
Translational Candidate Extracellular vesicles derived from stem cells for the treatment of corneal scars. Area of Impact The candidate aims to restoring vision by reducing the need for corneal transplant and the associated blinding complications. Mechanism of Action The therapeutic candidate is an extracellular vesicle (EV)-based therapy that could regenerate normal cornea tissue and reduce […]