Research Objective This research will discover whether transplantation of stem cell-derived microglia can be used to treat Sanfilippo syndrome, a devastating and currently untreatable childhood neurological disease. Impact If successful,…
Research Objective We will use integrated gene editing techniques to develop a new CAR-T cell therapy for multiple myeloma treatment Impact Develop an improved CAR-T cell therapy for patients with…
Research Objective We will develop a cell therapy for a rare skin disease. Patient-derived iPS cells will be genetically corrected and differentiated into epithelial sheets to be grafted on skin…
Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a…
Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As the…
Research Objective We are developing a tumor-associated glycan-targeting CAR T cell with inducible cytokine production that drives T cell stem/memory phenotype and persistence for effective treatment of ovarian cancer. Impact…
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…
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…
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…
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…
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