PEDIATRICS Vol. 92 No. 4 October 1993, pp. A48
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`GENE THERAPY' FOR GAUCHER'S DISEASE SHOWS PROMISING RESULTS IN TEST ON MICE

J. F. L. MD

University of Pittsburgh researchers reported promising results in mice with a potential "gene therapy" for Gaucher's disease that eventually may replace Ceredase, a Genzyme Corp. drug whose high cost has sparked controversy.

The study indicated it would be possible to transfer a gene into certain cells of people with the disease that would permanently correct a genetic defect underlying their disorder. The potential treatment, however, is at least several years away from widespread use in patients, and its development may face economic stumbling blocks.