The kids' immune systems now function well, a year after scientists transferred working copies of the gene into their bone marrow. The researchers warned everybody not to get too excited about short-term results, however. They won't be sure they have really cured the disease for many years. So the score stands at one probable success, a couple of ambiguous possible maybe successes, and that's about it. Penn's is just one of several investigations launched in the wake of Jesse's death, and their findings have been grim.
Gene therapy is in a heap of trouble.
The trouble is not just technical, it is also political. And much of it is self-inflicted.