409857
Position
Office of Tissues and Advanced Therapies Division of Cellular and Gene Therapies
Company
FDA/CBER
Bio
I received a Ph.D. in biochemistry from the University of Basel in Switzerland, after which I obtained training in virology and protein chemistry as an American Cancer Society Fellow in the Department of Biochemistry, Stanford University School of Medicine. While at Stanford I studied proteins encoded by Simian virus 40—work that supported development of the original Western blot technique. I also developed a novel chromatin immunoprecipitation assay that maps binding sites of specific DNA binding proteins on viral chromatin. In 1994, I joined the NIH/NINDS in 1994 to work on viral vectors for gene therapy, focusing primarily on the design of novel vector systems for transgene delivery into nondividing cells. I was among the first to design lentiviral vectors based on HIV-1. My paper was one of the three original reports that emerged on the subject in 1996. In 1999 I joined the LSU Health Sciences Center in New Orleans as an associate professor of medicine and to serve as the director of the LSU Gene Therapy Vector Core. My goal was to develop improved gene and protein transfer strategies for the CNS. My work dealing with lentiviral vectors and transgene delivery to the CNS was supported by R01 and R21 grants from the NIH, on which I was the PI. My research at CBER has focused on two of the major limitations of lentiviral vectors for clinical gene therapy, including the potential of such vectors to, 1) activate oncogenes during random integration into the host genome and, 2) transduce cells in off-target tissues in vivo. To address these shortcomings, I am developing safer HIV-1-based lentiviral vectors by, 1) limiting their integration to well-defined sites in the human genome, and 2) narrowing their tissue tropism through targeted transduction.