ISSN: ISSN: 2157-7412
Associate Professor, Department of Molecular and Human Genetics, Baylor College of Medicine
Philip Ng Associate Professor Department of Molecular and Human Genetics Baylor College of Medicine One Baylor Plaza Houston, TX, USA
My laboratory is interested in developing gene therapies for genetic diseases using helper-dependent adenoviral vectors (HDAd). HDAd (also called gutless or gutted adenovirus) do not contain any viral genes and thus represent a major improvement over early generation adenoviral vectors with respect to safety and efficacy. These vectors can transduce target cells with high efficiency to provide high level long-term transgene expression without chronic toxicity. Studies into improving the production of HDAd as well as their characterization are ongoing in my laboratory including manufacturing the vector under current Good Manufacturing Practices (cGMP) for clinical applications in humans. Another major focus of my laboratory is lung-directed gene therapy using HDAd with the primary goal of treating cystic fibrosis. In collaboration with Dr. Peter Hiatt, a pediatric pulmonologist, we have recently developed a novel method of aerosolizing HDAd into the lungs of nonhuman primates which has resulted in very high efficiency gene transfer to the airway epithelium with negligible toxicity. Based on these encouraging and compelling results, we plan to obtain regulatory approval from the FDA to use this novel technology to treat patients with cystic fibrosis in the near future.