Gene therapy for CF reaches human lungs.This week, researchers reported successfully transferring the normal version of the cystic fibrosis cystic fibrosis (sĭs`tĭk fībrō`sĭs), inherited disorder of the exocrine glands (see gland), affecting children and young people; median survival is 25 years in females and 30 years in males. (CF) gene to cells lining the lungs of a CF patient. This is the first step in an approach scientists hope will one day correct the underlying genetic defect causing CF. "This is very, very important in terms of demonstrating the feasibility of gene transfer for the treatment of cystic fibrosis," comments Robert J. Beall of the Cystic Fibrosis Foundation The Cystic Fibrosis Foundation (CFF) is a non-profit organization in the United States established to provide the means to cure and control cystic fibrosis. The Foundation provides information about cystic fibrosis (CF) and finances CF research that aims to improve the in Bethesda, Md. Last October, a team led by Michael J. Welsh of the University of Iowa Not to be confused with Iowa State University. The first faculty offered instruction at the University in March 1855 to students in the Old Mechanics Building, situated where Seashore Hall is now. In September 1855, the student body numbered 124, of which, 41 were women. in Iowa City reported inserting the healthy CF gene into cells lining the nasal passages of three people with cystic fibrosis (SN: 10/23/93, p.260). To achieve a treatment for CF, researchers had to figure out a way of getting this gene deep into the lungs. Scientists know this respiratory illness results when a mutant gene mutant gene n. A gene that has lost, gained, or exchanged some of the material it received from its parent, resulting in a permanent transmissible change in its function. causes the production of a defective protein. This flawed protein leads to a buildup of damaging mucus in the lungs. Ronald G. Crystal, former head of the pulmonary branch at the National Heart, Lung, and Blood Institute National Heart, Lung, and Blood Institute, n.pr established in 1948, this division of the National Institutes of Health is responsible for research and education on cardiovascular, pulmonary, systemic diseases, and sleep disorders. in Bethesda, Md., and his colleagues have forged ahead, inserting a healthy version of the CF gene into lung cells. Crystal, now at the New York Hospital-Cornell Medical Center in New York City New York City: see New York, city. New York City City (pop., 2000: 8,008,278), southeastern New York, at the mouth of the Hudson River. The largest city in the U.S. , and his coworkers relied on a modified adenovirus adenovirus Any of a group of spheroidal viruses, made up of DNA wrapped in a protein coat, that cause sore throat and fever in humans, hepatitis in dogs, and several diseases in fowl, mice, cattle, pigs, and monkeys. to get the healthy version of this gene into four CF patients. Each received a single dose of the therapy in the nose, followed by a single dose in the lungs. The researchers found that the healthy gene turned on in the nose cells of one patient and in the lung cells of another. They describe their findings in the September NATURE GENETICS. This preliminary experiment was designed to home in on safety issues. If found no evidence that the crippled adenovirus could replicate and thus cause infection. One patient in the study developed lung inflammation, a complication attributed to a reaction to the modified virus. This study proves that it's possible to get the healthy gene for CF into the lungs, Beall says. "It's another step along the way," Crystal says, noting that scientists still must show such therapy will ease the symptoms of this disorder. |
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