The odds that any son in that family would have the immune deficiency were gulp-worthy: 50-50. Nonetheless, the parents wanted more children to accompany the daughter they already had. And so they took a chance. They put their faith in the developing technology of bone-marrow transplants and the possibility that it had advanced far enough to build a sound immune system in a child who would lack one.
A possible new weapon in the medical arsenal is gene therapy, still in the clinical trial stage. With a harmless virus serving as the carrier, a healthy gene is inserted into a patient’s system to do the work of a defective gene that is the source of diseases like immune deficiency, sickle cell anemia and hemophilia. Gene therapy’s healing potential is widely accepted. But it has had plenty of setbacks along with triumphs since it was first tried in 1990, and has yet to be blessed by the Food and Drug Administration. Inevitably, too, there are ethical considerations. For some people, anything that smacks of genetic tinkering can touch off Frankenstein fears about whether we are redesigning human beings.
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