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Future 'patchwork' cure for hemophilia?

Future "patchwork' cure for hemophilia?

Researchers at the Fred HutchinsonCancer Research Center and the University of Washington, both in Seattle, are giving a genetic-engineering twist to basic procedures used in skin grafting. Using fibroblast cells infected with viruses carrying specific genes inserted in the laboratory, they hope to cure genetic disease with cell grafts.

Some genetic diseases are characterizedby a lack of certain substances, such as particular enzyme. According to the scientists, patches of fibroblasts that contain genes coding for the missing components may be induced to supply them, thereby reversing the disease.

Unpublished data presented by thescientists at last week's DNA/Hybridoma Congresses in San Francisco suggest this gene-therapy technique may have the potential to someday cure hemophilia, the genetic disease characterized by lack of a blood component essential to normal clotting. Because of this deficiency, hemophiliacs are susceptible to serious blood loss following minor cuts or tooth extraction.

While others are looking at tumorderivedfibroblasts as gene carriers, the Seattle group uses fibroblasts taken from normal individuals or from patients with the deficiency being treated. In vivo work has begun in rats and will later move to dogs. Also, because substances produced by the added gene may not effectively reach the plasma from a skin patch, other routes of fibroblast introduction are being studied.

On March 2, the Food and Drug Administrationannounced the approval of a new drug called tranexamic acid, which protects the weak blood clots formed in hemophiliacs undergoing surgery. Although the drug is an important step for hemophiliacs, its efficacy is affected by severity of the disease. Gene-therapy techniques like that being studied in Seattle may offer a better solution.

In addition, a report from the Seattlescientists in the February PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (Vol.84, No.4) describes a similar system that restored production of adenosine deaminase (ADA) in fibroblast cells from a patient with severe combined immunodeficiency syndrome. Without the ADA enzyme to destroy them, certain substances accumulate in the blood of ADA-deficient patients and cause immune system malfunction. Transfer of ADA-coding genetic material made the patient's fibroblasts produce 12 times as much ADA enzyme as was produced by cells from normal individuals.
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Author:Edwards, Diane D.
Publication:Science News
Date:Mar 14, 1987
Words:362
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