Breakthroughs in Gene Therapy for Hemophilia to Be Addressed at National Scientific Meeting Katherine High, M.D., of The Childrens Hospital of Philadelphia, to Describe Progress of Research in Gene Therapy for Hemophilia, Potential Applications to Other Diseases
Katherine A. High, M.D., director of hematology research at The Childrens Hospital of Philadelphia, is internationally prominent for clinical trials of gene therapy for the inherited bleeding disorder hemophilia, and an expert on the molecular biology of the disease.
Dr. High will deliver a Science Innovation Lecture, "Gene Transfer for Genetic Disease: Hemophilia as a Model for Prospects and Problems."
Friday, Feb. 15, 2002, 8:00 a.m., American Association for the Advancement of Science (AAAS) Annual Meeting, Sheraton Boston Hotel, Boston, Mass.
Gene therapy holds the potential of treating human disease at a fundamental level, by delivering therapeutic genes directly into a patients cells. In the first decade of this emerging field, from 1990 to 2000, researchers faced problems with efficiently delivering genes and achieving clinical improvement while avoiding toxic side effects. Advances in knowledge and technology may now be bringing success closer.
Dr. High will review her pioneering work on gene therapy for hemophilia B, including recent and current clinical trials with patients at The Childrens Hospital of Philadelphia and Stanford University. She will describe hemophilia gene therapy as a potential model for treating a wide range of human diseases.
The Children's Hospital of Philadelphia, the nations first childrens hospital, is second in the United States among all childrens hospitals in total research funding from the National Institutes of Health. For details, see www.chop.edu.