Disease Focus: Hemophilia A

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Genome Editing of Sinusoidal Endothelial Stem Cells for Permanent Correction of Hemophilia 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 permanent cure. Impact Permanent correction of hemophilia A by editing mutations in the FVIII gene in stem cells. Develop a precise and efficient non-nuclease genome […]

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

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 Our work would provide conceptual proof that a cell based therapy for hemophilia A is possible and should be pursued. Major Proposed Activities Demonstrate that […]