Modulating Liver Sinusoidal Endothelial Cell Permeability to Enhance Engraftment of Endothelial Cell Progenitors for the Treatment of Hemophilia A

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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…

Continue ReadingModulating Liver Sinusoidal Endothelial Cell Permeability to Enhance Engraftment of Endothelial Cell Progenitors for the Treatment of Hemophilia A

Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells

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Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how…

Continue ReadingGenome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells