Word: experimentally
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That obsession is gene therapy, and W. French Anderson, 57, more than anyone else, has brought it from the realm of science fiction to reality. It was Anderson who campaigned single-mindedly for the first approved test of the technology in 1990, who organized and supervised the trial, and who...
But Nelson Wivel, who is the head of the NIH's Office of Recombinant DNA Activities, defends the experiment. Anderson's detractors, he notes, "call him a zealot. But if it weren't for his zealotry, we probably wouldn't be doing gene therapy." Indeed, it was the ADA trial...
The most direct approach is to find a healthy copy of the missing gene and transplant it into the affected cells. That's the strategy Anderson, teaming up with Drs. Michael Blaese and Kenneth Culver at the National Institutes of Health, used in a landmark experiment three years ago. The...
Impressive as the experiment was, scientists knew that the girls had been treated but not cured. The altered blood cells died out after several months, and the patients had to return to the hospital periodically to repeat the procedure. To achieve a full cure, gene therapists would have to get...
And that's precisely what Blaese did -- only a week before the triumphant press conference last May. Going back to one of the original Ohio girls, he inserted healthy ADA genes into stem cells he had coaxed out of her bone marrow. He then inserted the altered cells into the...