Gene Therapy Used on Cholesterol Disorder : Medicine: The treatment has slowed the progression of heart disease in a woman with a rare malady.

Gene therapy has dramatically slowed the progression of heart disease in a woman with a severe inherited cholesterol disorder, and surgeons have already applied the technique to four other victims of the disorder, Pennsylvania researchers said Thursday.

Although the 30-year-old woman still has dangerously high cholesterol, the gene therapy probably will prolong her life, perhaps by years, said Dr. James Wilson of the University of Pennsylvania.

“I feel much better in the sense that I can do more physical activities now, such as skiing and dancing. I was limited before,” the seamstress and part-time bank teller from Quebec City, Canada, said Thursday.

The woman, who insisted on anonymity, has a severe form of a disease called familial hypercholesterolemia, in which the liver lacks the gene to produce a normally functioning protein that removes cholesterol from the blood. About one in a million people lack the gene.

Because of the disorder, cholesterol clogs the arteries of victims, most of whom die in their teens. The Quebec City woman had a heart attack at age 16 and underwent a coronary artery bypass graft at age 26.

Wilson’s team surgically removed about 15% of the woman’s liver. In the laboratory, they used viruses to shuttle copies of the missing gene into liver cells. About one-fifth of the cells took up the gene. The corrected cells were then put back into the woman’s liver.

“I had nothing to lose,” the woman said of the experimental treatment, whose results are reported today in the journal Nature Genetics.

In addition to helping the woman’s liver clear cholesterol from her blood, the gene therapy also made her more sensitive to the beneficial effects of a cholesterol-lowering drug called lovastatin, thereby increasing the overall efficacy of the procedure, Wilson said.