The University of Florida (UF) Division of Sponsored Research was awarded a $704,635 Project Grant under the National Heart, Lung, and Blood Institute's (NHLBI) Cardiovascular Diseases Research program (CFDA 93.837). The grant, titled "CAPSID- AND GENOME-MODIFIED AAV3 VECTORS FOR HEMOPHILIA GENE THERAPY," aims to develop optimized adeno-associated virus (AAV) vectors for gene therapy of hemophilia. Specifically, the project will evaluate the molecular mechanisms and in vivo efficacy of ITR-engineered AAV3 and AAV8 vectors in transducing human hepatocytes and correcting the phenotype of murine hemophilia models. The goal is to create more efficacious AAV vectors that require lower doses, thereby reducing immunogenicity and production costs to increase the probability of successful translation to clinical gene therapy applications for hemophilia B and A. The project will be conducted over a period of approximately 4 years, from the award date of December 15, 2024 through the ultimate completion date of November 30, 2028.
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