Gene therapy has been used to successfully treat a systemic disease – that is a disease that affects multiple organs and tissues throughout the body. Researchers at the University of Pennsylvania School of Veterinary Medicine, and Washington University School of Medicine used gene therapy to treat dogs with a condition called mucopolysaccharidosis VII, which is caused by a single defective gene. The condition also affects humans, and while it is treatable, the treatment is very expensive. The results of the study were promising and 17-months after treatment the dogs showed little evidence of the disease. Even though the study was carried out on dogs, the work is highly important as it is the first time gene therapy has ever been used to successfully treat a systemic disease. Study leader Dr Mark Haskins believes that the technique could be used to treat other systemic diseases such as hemophlia.
SOURCE/REFERENCE: Reported by www.bbc.co.uk on the 13th September 200
