The focus of our research is developing viral vector-based gene replacement strategies for the treatment of inherited ocular disease.
We research how the nervous system works and how it responds and repairs itself due to the damage caused by injury or disease. Our two main goals are to: Discover strategies for enhancing neuronal regrowth and staling neuronal loss after injury, and understand the nervous system response and recovery during damage caused by disease.
Dr. Hoffman is an Associate Professor and cellular immunologist with a major interest in immune modulation and tolerance induction using adeno-associated virus (AAV) gene therapy.
The Kang Laboratory focuses on the genetics of muscular dystrophy, and the core project involves gene discovery in limb girdle muscular dystrophy and other muscle diseases. The laboratory makes use of exome and genome sequencing technologies, supplemented as needed by linkage analyses and other approaches.
This lab is focused on developing new therapies of lysosomal storage diseases (LSDs) based on the understanding of molecular mechanisms of the pathogenesis of these diseases. These inherited metabolic conditions are caused by defects in a wide spectrum of lysosomal and a few non-lysosomal proteins resulting in accumulation of undigested substrates, resulting in dysfunction of lysosomal/endosomal system.
Our mission is to combine medical, scientific and technology skill-sets to discover new approaches to combat infectious disease outbreaks. We do this by listening to both patients and their healthcare providers to better prioritize problems and reveal unanticipated durable solutions.
The overall theme of the Pacak Lab research program is study and develop therapies for disorders that affect mitochondrial function. We apply mechanistic knowledge gained from rare disease studies to broader concepts such as the role of mitochondria in cardiac dysfunction and neurodegeneration that are highly relevant to many common health disorders.
Dr. Srivastava’s research has been focused on the following two parvoviruses, the non-pathogenic adeno-associated virus (AAV), and a common human pathogen, the parvovirus B19, and the development of recombinant parvovirus vectors for human gene therapy.
The primary mission is to merge molecular genetics research and health care by developing new therapeutic strategies involving gene transfer. The idea of gene therapy is a logical and natural progression of the last 20 years of research in medical genetics and molecular biology.
Powell Gene Therapy Center
The primary mission of the Gene Therapy Center at the University of Florida is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer. The idea of gene therapy is a logical and natural progression of the last 20 years of research in medical genetics and molecular biology.