Word: anderson
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...Anderson and Blaese are confident that gene therapy could fit the bill. Ideally they would prefer to insert ADA genes into bone-marrow stem cells, which would continuously manufacture blood cells containing the gene and ensure a steady supply of the enzyme. But researchers have yet to find a way to isolate marrow stem cells effectively. Instead, the NIH researchers opted for T cells, immune-system cells that can survive in the bloodstream for months and even years...
Extracting viable T cells from their young patient, Anderson and Blaese exposed them to mouse leukemia retro-viruses into which human ADA genes had been spliced. The retroviruses, rendered harmless by genetic engineering, were the vectors, the vehicles that would deliver the genes to their target. They invaded the T cells and, as retroviruses are wont to do, burrowed into the T- cell DNA, carrying the ADA gene with them. Finally, a billion or so T cells, now equipped with ADA genes and floating in the gray solution suspended above the little girl's bed in Bethesda, were dripped into...
...being "expressed" and that the enzyme is being produced. Even so, the child will not be cured. She will have to return monthly for at least two years to ) the NIH, where doctors will infuse her with more engineered T cells and check for possible side effects. But Anderson and Blaese are optimistic. "We're very comfortable with the concept," says Blaese. In the meantime, as a precaution mandated by the FDA, the girl will continue to receive the PEG-ADA drug treatment...
Rosenberg's strategy, devised with the help of Anderson, is to extract immune cells called tumor-infiltrating lymphocytes (TILs) from the tumors of melanoma patients. Rosenberg bathes the TILs in a solution of interleukin-2, a natural substance that invigorates them, and then exposes the TILs to re- engineered mouse leukemia retroviruses...
Like the retrovirus used by Anderson, Rosenberg's delivery system has been made harmless and endowed by recombinant DNA techniques with a human gene. But this gene codes for tumor necrosis factor, a naturally occurring compound that attacks cancer cells. The altered viruses insert themselves and their piggyback gene into the genetic material of the TILs, which are then injected back into the bloodstream of the melanoma patients. If everything goes as planned, the activated TILs will home in on the tumors like guided missiles, attacking the cancerous cells and at the same time releasing the antitumor factor to help...