Magazine article Science News

And Downs

Magazine article Science News

And Downs

Article excerpt

A few years ago, investigators found the broken gene that causes cystic fibrosis, the fatal disease that has since attracted the interest of many in the gene therapy field. By introducing working copies of the cystic fibrosis gene into the lungs of patients, physicians hoped to correct the mutation and prevent the mucus secretions that lead to infections in the lungs. According to animal studies and test-tube experiments with human cells, the most promising delivery vehicle for those genes was an adenovirus, a virus that infects lung cells.

In the most thorough test of this strategy in humans so far, however, investigators have found that adenoviruses are extremely inefficient at shuttling the cystic fibrosis gene into nasal cells with characteristics similar to those of lung cells. The viruses successfully delivered their cargo to less than 1 percent of nasal cells, report Michael R. Knowles of the University of North Carolina at Chapel Hill and his colleagues in the Sept. 28 New England Journal of Medicine. Higher doses of the adenovirus did not help: They simply irritated the patients' noses, the researchers note.

Gene therapy researchers argue that they are constantly developing better gene-carrying viruses and should be able to overcome these problems. "It's important to remember that gene therapy is truly in its infancy and that the current tools are quite crude," Jeffrey M. Leiden of the University of Chicago wrote in an accompanying commentary. …

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