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the far-out realms of astrophysics, with their time warps and black holes, there is a strange, other-world quality about gene therapy. At first, the idea itself is unsettling—that we might revise our genes, the biochemical basis of what we are. |
And consider the preferred messenger for this genetic revision—a virus. We know about viruses. Purveyors of infection and disease. Can this enemy really be redeemed? Can a virus be trusted to slip into our cells, toting its toolkit for genetic repair?
Even the experiments have a wonderland oddness about them. In the labs where treatments for cystic fibrosis are evaluated, the action very often revolves around an astonishing rodent—a mouse engineered with a nose that can mimic our own.
Of course, this sort of strangeness isn't new to science. Throughout history, the revolutionary breakthroughs, the theories and discoveries that would change the world forever, often seemed unreal. They messed with our minds. As Susan Navarette has found in her studies of 19th century horror, the theory of evolution set off monstrous anxieties and social shock waves that reverberate even today.
But for those who suffer from cystic fibrosis, or from hemophilia, or from any other deadly, inherited disorder, none of this strangeness matters. What the patients want to know about gene therapy is When? How much longer? And, Will I still be here when you get it right?
What impressed us most about Richard Boucher, Jude Samulski, and the other Carolina scientists who work in the field of gene therapy is that they manage to inhabit both worlds—the world of the patient and the world of the lab. This is a wrenching but necessary balance. It is hard to leave a clinic full of desperately ill patients, many of them children, and go back to a lab full of infinitely complicated, painstaking work. You can almost hear the patients say hurry. Hurry.
But that is exactly what these scientists know they cannot do. Yes, they will always be keenly aware of the goal—relief for the people who suffer. But if gene therapy is ever to work, the basic science will have to be done.
And they will have to get it right.