By Wilson da Silva
TORONTO – Canadian and American researchers who isolated the defective gene that causes cystic fibrosis said on Thursday their discovery had given new hope to all sufferers of the most common fatal genetic disease in North America.
The researchers said doctors would soon develop new drug therapies to treat cystic fibrosis based on what they discover from studies of the defective gene that causes it rather than blindly treating the symptoms.
“Drugs are beginning to be developed on the basis of our new-found knowledge,” Jack Riordan of Toronto’s Hospital for Sick Children told a news conference called to discuss the findings.
Riordan’s team and researchers from the University of Michigan, Ann Arbor, identified the defective gene and pinpointed the molecular defects that cause the disease in a majority of patients.
Canadian researchers Lap-Chee Tsui and Riordan of Toronto’s Hospital for Sick Children, together with Francis Collins, a Michigan researcher who headed the U.S. team, decided to hold the news conference after Reuters broke the story of their discovery on Tuesday three weeks before it was due to be published in the journal Science.
“Immediately, we can now go after therapy in a rational fashion, and in the long run, (identifying the gene defect) is an important step toward the goal of finding a cure for the disease,” said Riordan.
Researchers in Canada, the United States and Britain have been trying for years to isolate the gene for the disease, which afflicts at least 30,000 young people in the United States and dooms the sufferer to a short life marked by recurrent respiratory infections.
An estimated one person in 20 people in the United States and Canada unknowingly carries a copy of the defective gene and a child has a 25 per cent chance of inheriting the disease if both parents carry the defective gene.
Collins said the discovery would also lead to new opportunities for finding the genes causing other diseases and for finding possible cures.
“The success reported today gives a real sense of hope for cloning the genes of another 4,000 or so genetic diseases which affect mankind,” said Collins at the press conference.
“This milestone opens the door for new approaches in therapy,” said Collins.