Harvard stem cell researchers at Massachusetts General Hospital (MGH) have taken a critical step in perhaps making possible the discovery, in the relatively near future, of a drug to control cystic fibrosis (CF), a fatal lung disease that claims about 500 lives each year, with 1,000 new cases diagnosed annually. Beginning with the skin cells of patients with CF, Jayaraj Rajagopal, M.D., and colleagues first created induced pluripotent stem (iPS) cells, and then used those cells to create human disease-specific functioning lung epithelium, the tissue that lines the airways and is the site of the most lethal aspect of CF, where the mutant genes cause irreversible lung disease and inexorable respiratory failure. That tissue, which researchers can now grow in unlimited quantities in the laboratory, contains the delta-508 mutation, the mutation responsible for about 70 percent of all CF cases and 90 percent of the ones in the United States. The tissue also contains the G551D mutation, a mutation that is involved in about 2 percent of CF cases and the one cause of the disease for which there is now a drug. The work is featured on the cover of the April 6, 2012 issue of Cell Stem Cell. Postdoctoral fellow Hongmei Mou, Ph.D., is first author on the paper, and Dr. Rajagopal is the senior author. Dr. Mou credits learning the underlying developmental biology in mice as the key to making tremendous progress in only two years. "I was able to apply these lessons to the iPS cell systems," she said. "I was pleasantly surprised the research went so fast, and it makes me excited to think important things are within reach.
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