CYSTIC FIBROSIS CURE CLOSER

                         Roanoke Times
                 Copyright (c) 1995, Landmark Communications, Inc.

DATE: FRIDAY, January 10, 1992                   TAG: 9201100057
SECTION: NATIONAL/INTERNATIONAL                    PAGE: A-2   EDITION: METRO 
SOURCE: The New York Times
DATELINE: WASHINGTON                                LENGTH: Medium
CYSTIC FIBROSIS CURE CLOSER
Using a common cold virus like an overnight delivery service, scientists transferred a normal human gene that is missing in victims of cystic fibrosis directly into the lung tissues of laboratory rats.
Two days later, the animals' lungs were producing a protein that matched the gene. Without this protein, cystic fibrosis victims are condemned to early death.
The experiment was described as a significant step toward an effective treatment for cystic fibrosis, one of the two or three most common fatal genetic diseases among human beings.
"I have no doubt that if we could use this same system in humans, we could reverse the biological abnormalities in the lungs of people with cystic fibrosis," said Dr. Ronald Crystal, the government scientist who directed the rat experiment.
However, he cautioned that causing a normal gene to function in the lungs of laboratory rats is still a far cry from curing a disease.
"If I were talking to the parents of a child with cystic fibrosis and they asked me how long before this can be used to cure their child's illness, I'd have to say that I don't know.
"If we are lucky, we can begin human trials this year. I will say that I believe we will cure this disease. I believe there is a significant chance it won't be much longer."
There are about 30,000 cystic fibrosis patients in this country.
The genetic mutation leading to the disease was discovered less than three years ago. Victims produce a faulty version of a protein known as "CFTR," or cystic fibrosis transductance regulator.
The faulty version of CFTR fails to perform a key function in the process that enables people to clear mucus from the respiratory tract. Therefore, in persons with cystic fibrosis, the mucus remains in the lungs and harbors infections. Victims usually die as children or in early adulthood of lung damage from the infections.

by Archana Subramaniam by CNB