BETHESDA, Md. -- The first attempt to use gene therapy totreat a genetic disease appears to be working after eightmonths of treatment, National Institutes of Health scientiststold an NIH advisory committee here Friday.
The patient, a 4-year-old girl, suffers from a severe inheritedimmune deficiency disease. It is caused by lack of the gene thatproduces an enzyme called adenosine deaminase (ADA).Children afflicted with ADA deficiency, or "bubble boy's"disease, usually die of infection before they are 2 years old.
The child has received six infusions of her own blood, whichhas been genetically altered so that the white blood cellscontain the ADA gene.
The first six months of treatment increased the levels of ADAin the child's blood from less than 1 percent of normal to about20 percent of normal, said Michael Blaese, a co-researcher onthe gene therapy project. The child has had few side effectsfrom the treatment, he said.
"It's working, but is it working well enough? We're not doneyet," said Blaese. Blaese, together with co-researcher W. FrenchAnderson, intend to give the child two more infusions beforethey decide if the treatment is effective.
A second child is also being treated for ADA deficiency. --Rachel Nowak
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