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Valder R. Arruda, MD, PhD
Valder R. Arruda

The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research by Dr. Arruda. Working collaboratively, Dr. Arruda and his colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.

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Dr. Arruda is interested in the development of gene-based strategies for the treatment of bleeding and thrombotic diseases. In a collaborative effort, his 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 by Dr. Arruda 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. He is also identifying biological factors that modulate AAV vectors transduction and the risk of inadvertent germline transmission in animal models.