University of Iowa researchers this month published the results of their successful efforts using genetic engineering to prevent Cystic Fibrosis in mice.
Cystic fibrosis is the most common life-threatening genetic disorder in the United States — about 30,000 children and adults are affected by the disease. Due to a genetic mutation, salt does not get transported properly through the cells that line the lungs. This causes a thick build-up of mucus within the lungs and respiratory infections.
Although there have been numerous improvements in treatment for CF over the past couple decades, the median age of death for people with the disease is still around 25.
The new research conducted by Xiaoming Liu and colleagues in Iowa involved mice who received a graft of diseased human lung cells. The mice were then exposed to a virus which was engineered to implant a gene fragment into the diseased lung cells.
The gene fragment fools uses the process which the defective gene uses to create a new protein to slip in a corrected version of the gene in RNA. The mice exposed to the virus saw a 10 percent improvement in lung function which the researchers report was enough to keep the disease under control.
There are still a number of obstacles that will need to be overcome before this sort of approach could be tested in human beings, but it cannot be emphasized enough how animal testing has allowed researchers to make such significant headway into finding a cure for cystic fibrosis only a little over a decade since the gene defect that causes the disease was first discovered.
Source:
Gene therapy hope for cystic fibrosis. Jeremy Thomson, Nature, January 4, 2002.