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PITT GENETICISTS ACCOMPLISH GENE TRANSFER IN ANIMAL MODEL FOR GAUCHER DISEASE

 ~ADVANCE~ PITTSBURGH, Nov. 30 ~PRNewswire~ -- University of Pittsburgh researchers have used a retroviral vector in laboratory animals to transfer the gene needed to cure Gaucher disease.
 The study is published in the Dec. 1 issue of the Proceedings of the National Academy of Sciences.
 Gaucher disease, the most prevalent lysosomal storage disorder, results from deficient activity of the lysosomal enzyme glucocerebrosidase (GC). GC breaks down a fatty substance called glucocerebroside which is produced when old blood cells disintegrate. When not broken down, glucocerebroside accumulates in the spleen, liver, and bone marrow, causing the organs to malfunction. Approximately 20,000 Americans are affected by the disorder.
 "The results of this study strengthen the rationale for gene therapy as a treatment for Gaucher disease," said John Barranger, M.D., Ph.D., professor in the Pitt departments of human genetics and molecular genetics & biochemistry, and leader of the research team. Other Pitt researchers on the team include Paul Robbins, Ph.D., assistant professor of molecular genetics and biochemistry, and Sallie Boggs, Ph.D., associate professor of radiation oncology.
 The Pitt researchers used a replication-defective retrovirus to transfer the human gene for GC enzyme production into the bone marrow cells of mice, grew the cells in culture and then returned them to the mice after receiving radiation to eliminate their bone marrow. The enzyme was present in the blood supply of transplanted mice at four to seven months post-transplantation.
 To further test the ability of the transferred gene, the researchers performed secondary transplants on 22 mice using bone marrow cells from the long-term reconstituted mice. All 22 mice showed enzyme activity generated by the transplanted gene.
 "The results provided by this study demonstrate that macrophages, the cells produced by the bone marrow, in the transplanted animals repopulate the bone marrow, liver, spleen and lung, and manifest the human gene. This is an essential step toward testing gene transfer in human Gaucher patients," said Barranger.
 Barranger and collaborators at the Pittsburgh Genetics Institute and the University of Pittsburgh Medical Center are currently involved in the review process to gain approval for a human gene therapy trial involving Gaucher disease patients to begin in 1993.
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 ~CONTACT: Suzie Hunt or Kathia Kennedy of Health Sciences News Bureau, 412-624-2607, or fax at 412-624-3184~


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