Word: anderson
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Like the others gathered to mark the anniversary, Anderson paid tribute to Watson and Crick, whose accomplishment made all that followed possible. Watson was equally appreciative. "I just wish to thank everyone for being here," he said, "to help Francis and me celebrate what was really a very wonderful birthday party...
...Anderson, then at the NIH, with colleagues Dr. R. Michael Blaese and Dr. Kenneth Culver, extracted T cells from the little girl's blood and exposed them to a mouse-leukemia retrovirus that had been rendered harmless and endowed with a normal ADA gene. Invading the T cell, the retrovirus acted as a vector, depositing its genetic material, including the ADA gene, in the cell nucleus. After the re-engineered T cells were cultured, a process that produced billions of them, they were infused back into the child's bloodstream, where their new gene began producing the ADA enzyme...
...years after that historic experiment, Anderson reported to the Cold Spring Harbor symposium, both this child and another young Ohio girl who began the same treatment a few months later have acceptable levels of the ADA enzyme and are leading normal, healthy lives, needing only to return every six months for repeat treatments. This study, and one conducted by the University of Michigan's Dr. James Wilson on a woman with familial hypercholesterolemia, represent the only gene-therapy treatments to date with beneficial results. But Anderson expects more success from other projects getting under...
Other methods are more straightforward. In a forthcoming cystic fibrosis trial, Anderson says, doctors will simply "infuse the vector right down into the lungs. And there are even enemas of vectors for colon cancer...
Eventually, Anderson told his fellow Cold Spring Harbor celebrators, he looks to the day when "any physician can take a vial off a shelf and inject an appropriate gene into a patient...