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Penn Vet's Ophthalmology service is often in the news. That's because we have internationally renowned leaders in the field as our faculty and clinicians. Learn more about our team by reading some of the news articles below.
Creating an effective gene therapy for inherited diseases requires three key steps. First, scientists must identify and characterize the disease. Second, they must find the gene responsible. And finally, they must find a way to correct the impairment.
Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was born with a blinding disease. This ushered in a period of hope and progress for the field of gene therapy aimed at curing blindness, which culminated in the 2017 approval of a gene therapy that improved vision in people with Leber congenital amaurosis (LCA), a rare, inherited form of blindness closely related to the condition seen in Lancelot. It represents the first FDA-approved gene therapy for an inherited genetic disease.
Four faculty members at Penn Vet have been promoted to full professor in the Department of Clinical Sciences and Advanced Medicine: William A. Beltran, DVM, MSc, PhD; Margret L. Casal, DVM, Dr phil II, PhD; Wilfried Mai, Dr. Méd Vét, MSc, PhD; and Charles H. Vite, DVM, PhD.
Gene therapy holds promise for treating a variety of diseases, including some inherited blinding conditions. But for a gene therapy to be effective, one must know the precise gene responsible for a given individual’s disorder and develop a tailored treatment. For diseases that may be caused by mutations in many different genes, developing individual gene therapy approaches can be prohibitively costly and time-intensive to pursue.