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Principal Investigator

Dr Flotte received his undergraduate degree in the biological sciences from the University of New Orleans in 1982, and his medical degree from the Louisiana State University School of Medicine in 1986. After serving his residency in pediatrics at Johns Hopkins University, he completed a pediatric pulmonary fellowshipa and postdoctoral training in molecular virology there in 1992.

In 1995, Dr. Flotte and his colleagues at Johns Hopkins became the first to use adeno-associated virus, or AAV, as a vehicle to deliver corrective genes to targeted sites in the body, including the damaged airways of adults with cystic fibrosis.In 1996, Dr Flotte joined the faculty of the University of Florida and was appointed Associate Director of UF’s Powell Gene Therapy Center.

In 2000, he was named Director of the Powell Center and founding Director of the newly established UF Genetics Institute, a cross-campus multidisciplinary unit encompassing gene therapy, human genetics, agricultural genetics and comparative genomics. In 2002, Flotte stepped down from these roles to accept the position of Chair of the Department of Pediatrics.

An internationally known pioneer in human gene therapy, Flotte is currently investigating the use of gene therapy for genetic diseases that affect children, including cystic fibrosis, alpha-1 antitrypsin (AAT) deficiency, type I diabetes, and disorders of fatty acid oxidation. He is currently conducting Phase I trials with rAAV expressing alpha-1antitrypsin in AAT-deficient patients. He has also focused on the study of adeno-associated virus (AAV) vectors. Dr. Flotte's laboratory has also focused on the mechanisms of AAV persistence, since these represent the basis for a more profound understanding of the potential for long-term safe and effective gene therapy.

  

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