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PANEL OKS GENE THERAPY AS TREATMENT FOR CHILDREN

PANEL OKS GENE THERAPY AS TREATMENT FOR CHILDREN

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Gene therapy for human disease took a leap from laboratory theory to the real world of practicing medicine Wednesday as a National Institutes of Health panel approved a proposal to treat children with a severe genetic disease by inserting new genes into their blood cells.

The Institutional Biosafety Committee at the National Institutes of Health held the nation's first public hearing on a human gene therapy project and then unanimously approved it as safe.The project still needs the approval of six other federal regulatory panels, and the researchers are unlikely to begin clinical trials of the therapy before the end of this year.

But in weathering the scrutiny of the 12-member biosafety panel of scientists and local citizens, human gene therapy surmounted its first hurdle on the long regulatory road to the patient's bedside.

``As far as I know this is the first public review of a human gene therapy program ever,' said Dr. W. French Anderson of the National Heart, Lung and Blood Institute, an author of the therapy proposal.

Under the proposal, Anderson and Dr. R. Michael Blaese of the National Cancer Institute and their colleagues will try to treat young children suffering from adenosine deaminase deficiency.

ADA deficiency, a genetic disorder that destroys their immune systems, afflicted the famous ``bubble boy' from Texas, who had to live in a sterile plastic enclosure as a protection against infection.

Researchers believe human gene therapy may also prove valuable in the battle against cystic fibrosis, familial cholesterol disorders, metabolic deficiencies of the liver and other genetic disorders.

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