Gene Therapy Study For Gaucher Disease

Researchers at the University of Pittsburg Medical Center, USA, in cooperation with Genzyme Corp, have initiated a study of gene therapy in a 48-year-old woman with Gaucher disease, an inherited, life-threatening disorder that affects 10,000 to 20,000 in the USA alone. UPMC research team leader John Barrington said: "we are at the beginning of being able to develop a permanent solution to problems associated with Gaucher disease."

Currently, Gaucher disease is managed with enzyme replacement therapy, developed at Dr Barrington's laboratory while he was at the National Institutes of Health, and this was commercialized by Genzyme, which has licensed the technology from the University. In the latest research, five patients are to receive GM-CSF, which increases the production of specialized white blood cells; stem cells will then be removed and exposed to a retrovirus.