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…
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…
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…
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…
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…
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan…
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 Therapeutic candidate to cure hemophilia A is AAV-based genome editing vector that corrects the disease-causing mutation in the factor VIII gene in patient stem cells to develop a…
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 We propose to develop a lead small molecule inhibitor of PTPσ, a receptor expressed by human blood stem cells, for the purpose of promoting human hematopoietic regeneration. Impact…
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