choice of the litter. Some of these piglets are born with CF-like disease.
C. Rogers et al., Science
The gene responsible for cystic fibrosis (CF) was discovered there near 20 years, but since then progress to conquer this devastating lung disease has been slow. Now researchers have a new tool designed to develop pigs CF They are considered a breakthrough because they are born with the disease features as those observed in infants, while mouse models are not.
Approximately a 4,000 babies in the United States is born with CF. It is caused by a defect in the gene CFTR , which generates an ion channel protein that moves salt and water across the cell membrane in certain tissues. The lungs of CF patients are normal at birth, but over time become clogged with sticky mucus and becomes vulnerable to infection; despite new treatments, many patients die in their 20s or 30s. The mouse created for the CFTR default are far from the human reality of the disease: They do not develop disease or lung injury to other organs such as the pancreas. And it also means they do not provide much insight into how the disease progresses in children. So Michael Welsh, physician and molecular physiologist at the University of Iowa in Iowa City, sought a better animal model "frustration," he said.
thereFive years, Welsh teamed with animal scientist Randall Prather of the University of Missouri, Columbia, which develops transgenic pigs ( science NOW, January 2, 02). They have disabled the first CFTR existing gene in pig cells then cloned pigs from these cells, which was not easy because the two stages have been developed that in mice. Last April, the researchers reported these findings in Journal of Clinical Investigation , describing the pigs with a silent copy of CFTR . they then mated those pigs to see if the descent with two bad copies of CFTR to develop a CF-like disease. "We did not know if we were to end up with a big mouse," Welsh said.
Instead, they got something much closer to a human with CF, reports of the Welsh team in this week's issue of science . The newborn piglets have many of the same signs of CF observed in infants, including transportation impaired salt, intestinal blockages and pancreas and liver damage. - But normal lungs
One difference is that, although only 15% of infants are born with an intestinal blockage, all pigs were, and many had to be euthanized young. The team performed a surgery to correct the blockage on some pigs and watching as they grow for signs of lung problems. "We're excited, but we're still holding our breath," said Welsh. He and several others founded a company that will sell the pigs to pharmaceutical companies and researchers studying FC
Others in the area are CF announce the arrival of pork. "It is a remarkable seminal achievement," said Eric Sorscher of the University of Alabama, Birmingham. He and others say it should be useful not only for the study of the natural history of the disease, but also for the improvement of gene therapy for cystic fibrosis, which has had limited success. "it is a beautiful piece of work and it's good to see happen," confirms Richard Boucher of the University of Carolina North, Chapel Hill.
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