The combination of molecular cloning and development of powerful gene transfer technologies has enabled us to generate novel molecular medicines to treat genetic diseases. Importantly, gene therapy can provide lasting treatment and perhaps even cure a disease after a single round of administration of the vector or of gene-modified cells. Gene therapy is therefore well positioned to eventually replace more conventional treatments that require frequent drug injections.
Our laboratory is primarily utilizing adeno-associated viral (AAV) vectors to transfer therapeutic genes for the correction of the bleeding disorder hemophilia. In addition, we study the immune response to gene therapy to ensure that a patient’s immune system does not reject the therapy. Finally, we are developing innovative technologies and protocols to induce immune tolerance to ensure lasting therapy.
This multidisciplinary approach to research (including molecular biology/recombinant DNA, virology, hematology, immunology, genetic engineering, and other disciplines) fuels exciting collaborations between our group and multiple other laboratories at the University of Florida and elsewhere and unique opportunities for the training of students and postdoctoral and clinical fellows to be prepared for the challenges of future and ever more complex biomedical research.