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2004 PC Project Grant Award Dennis Roop, Ph.D. and Jiang Chen, MD Baylor College of Medicine Project Title: Generating an Inducible Mouse Model for Pachyonychia Congenita
Biography Dennis Roop received a B.A. degree in Biology from Berea College, Berea, KY in 1969 and a M.S. and Ph.D. in Microbiology from the University of Tennessee, Knoxville, TN in 1972 and 1977, respectively. He was a postdoctoral fellow with Dr. Bert O'Malley at Baylor College of Medicine, Houston, TX, from 1977 to 1980. Subsequently, he joined the laboratory of Dr. Stuart Yuspa at the National Cancer Institute, National Institutes of Health in Bethesda, MD. In 1988, he was recruited back to Baylor College of Medicine, where he is currently Professor of Molecular and Cellular Biology and Dermatology and Director of the Center for Cutaneous Molecular Biology.
His research interests have focused on the molecular mechanisms regulating skin and appendage development for more than twenty years. He is the recipient of several awards including: a Max Plank Research Award from the Alexander von Humboldt Foundation (1991), the William Montagna Lecture Award from the Society for Investigative Dermatology (1992), the Michael E. DeBakey, M.D., Excellence in Research Award (2001), the CE.R.I.E.S. Research Award (2002) and the Tanioku Kihei Memorial Lecture Award from the Japanese Society for Investigative Dermatology (2003).
He has served on the Advisory Council at the National Institute of Arthritis and Musculoskeletal and Skin Diseases and he is currently President of the Society for Investigative Dermatology.
Jiang Chen received his bachelors of medical science from the Henan Medical University in Zhengzhou China before obtaining his M.D. degree from the University of Heidelberg in Germany. His post-doctoral training began at the University of Tuebingen in Germany and is currently continuing in the Department of Dermatology at Baylor College of Medicine in Houston, Texas with Dr. Dennis Roop.
His research experiences have included cloning a therapeutic gene, preclinical work toward development of gene therapy of diabetic wound healing and cooperative development of a therapeutic vector with a German company.
Abstract Mutations in keratin genes have been identified in individuals with PC. Embryonic stem cell technology has allowed the generation of mouse models that mimic human diseases. These mouse models are valuable tools for the development and evaluation of novel therapeutic approaches for human diseases. Unfortunately, it is not possible to establish mouse models for all inherited diseases. Newborn mice genetically engineered to reproduce inherited forms of skin fragility syndromes often die within a few days of birth due to uncontrolled water loss and/or infections.
To overcome this problem, we have recently developed an inducible system that allows the activation of mutations in a restricted area of the skin. Besides symptomatic care, no effective therapeutic treatment is available for PC patients. Therefore, gene therapy may be the only option for a permanent corrective therapy for these patients. Prior to testing gene therapy for PC in humans, it is desirable to utilize a preclinical animal model to determine the efficacy and safety of these approaches. Therefore, we propose to develop a “humanized” inducible mouse model that represents PC-1 at both the genetic and phenotypic levels. This mouse model will serve as an ideal animal model for preclinical testing of gene therapy and other novel therapeutic approaches for PC.
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