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The Arruda Lab is interested in the development of gene-based strategies for the treatment of bleeding and thrombotic diseases. In a collaborative effort, the lab and others have carried out early-phase clinical studies on adeno-associated viral (AAV) vectors for the treatment of severe hemophilia B (factor IX deficiency).
Current projects in the Arruda Lab are focused on translational research studies on the efficacy and safety of intravascular delivery of AAV vectors to skeletal muscle or liver of life models with severe hemophilia. The lab is also identifying biological factors that modulate AAV vectors transduction and the risk of inadvertent germline transmission in animal models.