PHILADELPHIA -- For the first time, researchers have successfully used gene therapy to treat a disease that affects organs throughout the body of a large animal. The results will be reported this week on the Web site of the Proceedings of the National Academy of Sciences. The study, led by Mark E. Haskins of the University of Pennsylvania School of Veterinary Medicine and Katherine Parker Ponder of Washington University School of Medicine in St. Louis, involved dogs with a rare disorder in which an enzyme deficiency causes clouding of the corneas, cardiac disease and bone abnormalities leading to loss of mobility by six months of age. Newborn dogs treated with hepatic gene therapy maintained near-normal mobility throughout the 17-month study and showed little evidence of the other debilitating signs normally associated with the disease.
The above story is based on materials provided by University Of Pennsylvania. Note: Materials may be edited for content and length.
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