Human Stem Cell Use: Adult or Tissue Stem Cell


A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells

Research Objective A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells Impact We aim to develop a novel genome editing based therapy for Artemis-deficient severe combined immunodeficiency that would improve upon prior gene therapies in efficacy, safety, and scalability. Major Proposed Activities Discover optimal approach for nonviral […]

Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells.

Research Objective Novel selective pharmacological strategy targeting senescent lung stem cells Impact Idiopathic pulmonary fibrosis along with other interstitial and age-related lung diseases Major Proposed Activities Chracterize senescence cells, including senescent stem and progenitor cells in the lung of patients affected by idiopathic pulmonary fibrosis Screening of a Rubedo Life Science library of senolytic small […]

An hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies.

Research Objective We propose to transduce hematopoietic stem cells with vectors that encode chimeric antigen receptors targeting HIV for T cells and anti-HIV broadly neutralizing antibodies for B and/or plasma cells. Impact Recent methods are limited by the rise of escape mutants against a single CAR. Our approach solves this issue by the ability to […]

Combating Ovarian Cancer Using Stem Cell-Engineered Off-The-Shelf CAR-iNKT Cells

Research Objective HSC-engineered allogeneic mesothelin-targeting CAR-iNKT (AlloMCAR-iNKT) cells Impact treatment of ovarian cancer Major Proposed Activities Milestone 1. Production of the AlloMCAR-iNKT cells Milestone 2. Characterization of the AlloMCAR-iNKT cells Milestone 3. Delivery of the new therapeutic candidate Ovarian cancer (OC) is the leading cause of death among women with gynecological malignancies. In the USA, […]

Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)

Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This application will enable development of a therapeutic candidate for the treatment of Myelodysplastic Syndromes, a preneoplastic hematological condition of HSCs. Major Proposed Activities Determine the […]

Defining the Optimal Gene Therapy Approach of Human Hematopoietic Stem Cells for the Treatment of Dedicator of Cytokinesis 8 (DOCK8) Deficiency

Research Objective A new therapeutic option for DOCK8 deficiency using autologous human hematopoietic stem cells modified through either lentiviral gene addition or CRISPR/Cas9 based gene editing. Impact Allogeneic HSCT is complicated by comorbidities that can be addressed by autologous stem cell gene therapy. This is relevant for DOCK8 deficiency and can be applied broadly to […]

Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia

Research Objective The objective of this research is to define the final therapeutic candidate for effective hematopoietic stem cell gene therapy to treat severe alpha thalassemia that requires life-ling transfusions Impact Severe alpha thalassemia may lead to fetal demise or a life-long need for chronic transfusions with multiple medical complications, especially iron overload from transfusions. […]

Targeted Immunotherapy-Based Blood Stem Cell Transplantation

Research Objective An engineered antibody construct that targets and recruits immune cells to kill diseased blood stem cells, including leukemia stem cells, so that healthy stem cells can replace the diseased ones Impact An antibody that can direct immune cells to kill diseased stem cells would make stem cell transplant safer, more accessible and effective […]

Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis

Research Objective Locally delivered formulation of RSpo1 protein as an activator of Lgr5+ epithelial stem cells in chemotherapy- or radiation therapy-induced oral mucositis Impact Oral mucositis Major Proposed Activities RSpo1 formulation design and selection for optimal oral delivery Activation of Wnt pathway by formulated RSpo1 in-vitro Production of RSpo1 protein Oral stem cell expansion by […]

Development of a novel stem-cell based carrier for intravenous delivery of oncolytic viruses

Research Objective Develop a stem cell-based platform that safely and efficiently delivers viruses that specifically kill tumor cells and restore immune activity in patients with advanced cancer. Impact Overcome the inherent limitations that prevent efficient intravenous delivery of tumor killing viruses to metastatic tumors. Major Proposed Activities Compare the relative efficacy of native or cargocyte-delivered […]