Hundreds of scientists are looking to gene therapy to help cure intractable diseases, including the following:
• Cystic fibrosis. The most common deadly inherited disease among Americans, cystic fibrosis affects those who lack a gene to make the chemical that causes salt to move beneficially in and out of the cells. As a result, phlegm clogs the lungs of victims, drowning them. They often die in their teens.
At the National Institutes of Health in Bethesda, Dr. Ronald Crystal gave gene therapy to four cystic fibrosis patients in their 20s. To solve the salt problem, he used a common cold virus to carry the missing gene into their lung cells. Their condition is stable, but it’s too early to tell, said Dr. Crystal, who now heads the Division of Pulmonary and Critical Care Medicine at New York Hospital. Still, he says, “This has the potential to cure cystic fibrosis.”
• Familial hypercholesterolemia. This rare disease affects people who lack a gene enabling the liver to regulate and store cholesterol. As a result, cholesterol piles up, sticking to the walls of blood vessels, clogging or narrowing them, and impeding blood flow. Patients often die of heart attacks before their teens. Dr. James Wilson, director of the Institute for Gene Therapy at the University of Pennsylvania in Philadelphia, grew the liver cells of three such patients in lab dishes and sprinkled the cells with a virus spliced with the missing gene. He then injected the patients with their altered liver cells. His first subject was a woman of 26 who’d had her first heart attack at age 16. “She has had no problems since the procedure,” says Dr. Wilson. “She is not totally cured but is significantly improved.”
Questions have been raised. For example, if scientists found genes for intelligence and used gene therapy on early embryos to produce more intelligent children, would it be moral? “The ‘designer child’ is way down the road,” said Arthur Caplan, director of the Center for Biomedical Ethics at the University of Minnesota. “But, in 50 years, I’d be shocked if we weren’t in debate about designing our descendants.”
There may be more questions than answers now, but scientists know gene therapy is giving them a handle on something earthshaking. They are not about to let it go.
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