Genetic Breakthrough Seen in Hunt for Cystic Fibrosis Cure

Scientists said Thursday that they have wiped out certain cystic fibrosis symptoms in mice, an advance in the search for a cure for the inherited disease that usually kills its human victims before their 30th birthdays.

The research, being published today in the journal Science, establishes for the first time that the disease in mice can be corrected with a human gene. The work focused on intestinal symptoms and researchers are now probing whether a similar approach will work on mice lungs.

Researchers took a normal copy of the cystic fibrosis gene, and inserted it into intestinal cells of mice. These mice had been genetically altered to show characteristics of cystic fibrosis, including the type of lethal intestinal blockage that strikes human babies with the disease.

When the normal gene was inserted, the intestinal symptoms cleared up.

“The mice . . . lived a normal life span,” Whitsett said, even though they would have died within a month without the genetic intervention.

The normal gene did not have to be inserted into every affected cell to show these benefits. Treating just one section of the intestine was sufficient.

Though the research strongly suggests a cure is feasible, it still remains years away, Dr. Jeffrey Whitsett, director of pulmonary biology at Children’s Hospital Medical Center in Cincinnati and the lead author of the Science article, said in a telephone interview.

Cystic fibrosis strikes the lungs, pancreas and intestinal tract, and is the most common genetic disorder among Caucasians, affecting 1 out of 2,000. People who have the disease cannot transfer salt and water normally across the cells of their lungs and other organs. Thick mucus secretions accumulate, clogging the airways of the lungs.