Gene Therapy for Cystic Fibrosis Patients

By Ezzell, Carol | Science News, December 12, 1992 | Go to article overview

Gene Therapy for Cystic Fibrosis Patients


Ezzell, Carol, Science News


Three teams of scientists received approval last week from a national panel of physicians and ethicists to administer gene therapy to a limited number of adults with cystic fibrosis -- the most common lethal hereditary disorder in the United States.

The National Institutes of Health's Recombinant DNA Advisory Committee (RAC) voted to allow each team to squirt solutions of genetically engineered cold viruses into the nasal passages and lungs of patients. The scientists hope the viruses will ferry corrected copies of the defective gene that causes cystic fibrosis into patients' airways, reducing production of the thick, lung-clogging mucus characteristic of the disorder.

Cystic fibrosis is most prevalent among whites, striking one of every 2,000 white children born in the United States. Patients experience recurrent lung infections and difficulty breathing because of mucus accretions in the lungs. In some patients, mucus builds up within the pancreas, intestines, or sperm ducts as well, leading to nutritional deficiencies or male sterility

There is no effective treatment. for cystic fibrosis, although physicians are testing several mucus-dissolving drugs. Most patients rely upon daily physical therapy to dislodge lung mucus and antibiotics to prevent infections from occurring within mucus-clogged organs. Nevertheless, few individuals with the disorder survive past age 30.

For years, the exact cause of cystic fibrosis remained a mystery Most researchers suspected it had something to do with the body's regulation of salt, because people with the disorder have abnormally salty sweat. Within the past three years, however, researchers have identified the gene that, when damaged, causes cystic fibrosis (SN: 9/2/89, P. 149). They have also found that the gene -- called the cystic fibrosis transmembrane conductance regulator (CFTR) -- normally codes for the production of a tunnel-like protein through which cells excrete salty chloride ions (SN: 3/2/91, p. 132). When mutated, the gene produces a protein that fails to do the job, creating instead conditions favorable to mucus deposition.

In the new gene therapy experiments, researchers will attempt to correct this defect by inserting functional CFTR genes into cystic fibrosis patients. The three research teams -- led by Ronald G. …

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