Altered Stem Cells Offer Cystic Fibrosis Hope

Patients with cystic fibrosis could one day be treated using their own stem cells which have been given a shot of gene therapy to fix a faulty gene, suggests a new study. US scientists first used a virus to ferry into stem cells the correct version of a gene which is faulty in cystic fibrosis. They then coaxed the stem cells, derived from human bone marrow, into becoming epithelial cells which line the airways of the lungs. In the test tube, these corrected cells appeared to function better than the uncorrected ones. "Our results provide a proof of principle that a cell-based therapy using marrow stromal stem cells is both a feasible and promising clinical approach," says Jay Kolls at the University of Pittsburgh, US, who led the study. "We are hopeful that we can perform a small clinical trial within the next two to three years." This is the first time the two new technologies have been combined in an attempt to tackle cystic fibrosis, says Bruce Bunnell, another team member, at Tulane University in New Orleans, US. "The results have provided us with a great amount of hope that maybe we can intervene in this horrible disease." Stem cells are primitive cells which have the potential to develop into a number of tissue cells in the body. The advantage of using a patient's own stem cells to repair or replace tissue is that they do not invoke an immune reaction.