Veterinarians, physicians and scientists at the University of Pennsylvania, School of Veterinary Medicine and Scheie Eye Institute and the University of Florida collaborated on novel gene therapy in dogs for a blinding disease called autosomal dominant retinitis pigmentosa. The group generated a gene therapy construct that introduced genetic material into cells to compensate for abnormal genes or to make beneficial rhodopsin that appears to have application for humans.
- William Beltran, DVM, PhD
- Gustavo Aguirre, VMD, PhD
- Artur Cideciyan, PhD
- Samuel Jacobson, MD, PhD
- Alfred Lewin, PhD
- William Hauswirth, PhD
Three key benefits of the findings
- A canine model for a common and severe form of X-linked retinal degeneration demonstrated that gene therapy successfully stopped photoreceptor cell death and prolonged vision.
- Results suggest substantial potential and validity in treating and evaluating animals with mid and late-course disease in other neurodegenerative disorders relevant to humans.
- The study demonstrates the importance of establishing proof of concept in animal models that truly recapitulate the stages of disease of human patients enrolled in phase II/III clinical trials.