Gene therapy promising for Fabry disease

Nov. 15, 2004 at 8:28 PM
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TORONTO, Nov. 15 (UPI) -- University of Toronto researchers have developed a gene therapy that shows promise for early and sustained correction of Fabry disease.

Fabry disease is an inherited disorder whose sufferers have a life expectancy of only 40 to 50 years and it is one of about 40 lysosomal storage disorders that collectively affect approximately one in 7,700 people.

Due to the genetic defects, those with the disease have a buildup of lipids or fats in endothelial cells lining vessel walls, causing constriction, which ultimately results in cardiac disease, kidney disease or stroke.

"Patients with Fabry disease either don't make enough of the enzyme alpha-galactosidase A or improper synthesis means the enzyme isn't active enough," said Jeffrey Medin of the University of Toronto and the Ontario Cancer Institute at the Princess Margaret Hospital.

In the study, published in the the Proceedings of the National Academy of Sciences, Medin demonstrated an engineered lentiviral vector -- a type of retrovirus -- administered a day or two after birth can correct the defect long term in mice with Fabry.

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