BETHESDA, Md.--A healthy copy of the cystic fibrosis (CF) genecan be transferred into the lung epithelial cells of the cottonrat, researchers reported here Friday. The results lend hope tothe possibility of gene therapy to treat CF, the most commonlethal genetic disorder among whites.
"Clearly, gene therapy to treat cystic fibrosis is feasible," saidRonald Crystal, chief of the National Heart, Lung and BloodInstitute (NHLBI), based here, and the leader of the researchteam.
CF is caused by mutations in the gene encoding for a proteindubbed CFTR. Faulty copies of CFTR block the transport of ionsacross cell membranes in the lungs and other tissues. Thisresults in the buildup of a thick mucus in the lungs, causing thechronic lung infections from which most CF sufferers die whilein their 20s. By using gene transfer to replace the faulty genesin a CF sufferer's lungs, researchers hope to eventually providea cure for a disease.
The CF gene was introduced into a rat lung cell. Beforeprogressing to human trials, Crystal warned, researchers willneed to prove safety in animal trials showing that theintroduction of genes in adenoviruses does not damage the lungepithelium tissue; that the gene goes only to cells requiringcorrection; and that it is not over-expressed, leading to otherion transport problems. -- Rachel Nowak
(c) 1997 American Health Consultants. All rights reserved.