CF gene therapy on horizon.

In a large-scale study, scientists have inserted healthy copies of the human gene associated with cystic fibrosis into rodent lung cells. Experimental gene therapy for humans might start as early as a year from now, says Ronald G. Crystal of the National Heart, Lung, and Blood Institute in Bethesda, Md.

IN 1989, a U.S.-Canadian team identified the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which, when faulty, leads to cystic fibrosis (SN: 9/2/89, p.149). Last spring, Crystal reported using an altered cold virus to insert a healthy human CFTR gene into the lung cells of three cotton rats (SN: 3/2/91, p.132). The virus can penetrate airway cells of the cotton rat, a cross between a rat and a hamster.

Crystal's team reports in the Jan. 10 CELL testing the technique on hundreds of cotton rats and successfully inserting the healthy human CFTR gene into epithelial cells lining the animals' lungs. Once inside the epithelial cells, the human gene turned on, producing its protein product for at least two weeks, Crystal says.

"This is a direct strategy that has the potential of curing the disease," he told SCIENCE NEWS.