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…
Research Objective CRISPR/Cas9 mediated FOXP3 gene editing in patient-derived hematopoietic stem cells as a cure for IPEX syndrome Impact FOXP3 mutation in IPEX syndrome leads to immune system dysregulation. Allogeneic…
Research Objective We aim to develop conditions for stable expansion of blood stem cells outside of the body Impact Blood stem cells are a rare but necessary cell type for…
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create…
Research Objective We aim to demonstrate that regulators of endothelial cell permeability can foster engraftment of endothelial cell progenitors in the liver sinusoids leading to production of Factor VIII. Impact…
Research Objective We will develop a new agent that can increase the production of hematopoietic stem and progenitor cells and determine how the compound functions Impact We aim to develop…
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…
Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells…
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