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Scientists Preserve Vision in Dogs with a Disease Similar to Retinitis Pigmentosa in Humans

Veterinarians, physicians and scientists collaborated on novel gene therapy in dogs for a blinding disease called autosomal dominant retinitis pigmentosa.

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.

INVESTIGATORS

  • 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.
Scientists Preserve Vision in Dogs
Dose–response function and long-term durability of gene therapy intervention at initial disease stage. Treated loci (green) remain near wild-type thickness (thin dashed lines), whereas untreated loci (red) show progressive thinning along the trajectory expected from the natural history of disease (thick dashed lines). PNAS 10/2015, 112 (43) E5844

LINKS

Gene therapy staves off blindness from retinitis pigmentosa in canine model
Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease