Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy

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Research Objective A vaccine booster to induce antigen-specific T memory stem cells that will help enhance the long-term immunity against cancer recurrence Impact Cancer recurrence presents an unmet medical need.…

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

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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…

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

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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…

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

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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…

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

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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 cellsMilestone 2. Characterization of the AlloMCAR-iNKT cellsMilestone 3.…

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

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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…

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