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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CAR T cells targeting abnormal N-glycans for the treatment of refractory/metastatic solid cancers

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Research Objective Develop genetically modified chimeric antigen receptor T cells to kill incurable solid cancers by targeting a previously un-targetable tumor associated carbohydrate antigen. Impact Refractory/metastatic solid cancers are almost…

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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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Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models

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Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini…

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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