Med Center dedicates gene therapy center

The establishment of the F.M. Kirby Center for Molecular Ophthalmology at the Medical Center promises to distinguish the University as a leader in the field of gene therapy. The $5 million center, located in a new biomedical research building on the Medical School campus, is the first molecular biology research facility devoted exclusively to the genetic treatment of vision loss. "It will have wide ramifications for both genetic science and the care of patients with hereditary diseases," said William Kelly, chief executive officer of the Medical Center and dean of the School of Medicine, in a speech at the dedication of the Center last Saturday. Gene therapy involves injecting a normal gene into a person to replace or block the functioning of a defective one, Kelly added. Scientists and researchers at the Center are currently studying eye diseases such as retinitis pigmentosa, macular degeneration and hereditary cataract, according to Stuart Fine, chairman of the University's Department of Ophthalmology and director of the Scheie Eye Institute. In many of these diseases, researchers claim, the deterioration of the retina occurs due to the inheritance of a defective gene or genes, causing blindness. "It is now possible by means of analysis of family trees and blood samples to understand exactly what the molecular abnormality [which causes retinal degeneration] is," Fine said. "What we really hope to do in conjunction with the Institute for Human Gene Therapy at Penn is to identify the specific molecular defect and then do gene therapy in some patients in order to correct that defect," he added. The Medical Center established the Institute for Human Gene Therapy two years ago under the direction of James Wilson. Within months, Wilson and his team set up unprecedented clinical trials for cystic fibrosis, Kelly said. "The Kirby family gift assures that this work will be extended to ophthalmology in a significant way," he said. At the dedication, scientists discussed the most recent findings in their research. Jean Bennett, assistant professor of ophthalmology, said her research has focused on retinal degeneration in the eyes of mice. Her team has injected genetic DNA material into their eyes in hopes of restoring their vision. "I think that it is a very timely decision to have a center that is devoted to the molecular side of ophthalmology because of all the new discoveries of gene mutations that cause blindness," said Dean Bok, professor of ophthalmology at UCLA, who also spoke about developments in retinal degeneration research. "I think it is a very important step -- a very exciting time for ophthalmology," he added. Kelly said the University has had a distinguished 124-year history in ophthalmology. In fact, the medical faculty's first specialists were two ophthalmologists who came to the University in 1870. Fred Kirby, the center's donor, said he is looking forward to hearing about the developments coming out of the center. "My expectations are that the new research facilities are going to produce scientific advances with respect to the eye that cannot be visualized at the present time," said Kirby. "I just have confidence that the lab is both well-conceived and headed by very capable people."