Health See Inside A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment Fundamental understanding of basic biology has set the stage for new treatments for cystic fibrosis By Steven M. Rowe, J. P. Clancy and Eric J. Sorscher Illustration by John Hendrix In 1989 when scientists discovered the defective gene that causes cystic fibrosis, a serious hereditary disorder that primarily strikes children of European descent, it seemed as though a long-hoped-for cure might soon follow. After all, tests in many laboratories showed that providing normal copies of the gene should enable patients to make healthy copies of the protein specified by the gene. If successful, that feat would go a long way toward restoring health in the tens of thousands of people around the world who suffered from cystic fibrosis and typically died in their late 20s. (Half of all patients now live to their late 30s or beyond.) The question was whether researchers would be able to reliably insert the correct gene into the proper tissues in patients’ bodies to rid them of the illness forever. That task proved harder than anyone had believed. Although scientists successfully engineered viruses to ferry copies of the correct gene into patients’ cells, the viruses did not do the job well. By the late 1990s additional unexpected complications made it increasingly obvious that another approach to addressing the fundamental problem in cystic fibrosis would need to be found. This is only a preview. Get the rest of this article now! Select an option below: Buy Digital Issue Customer Sign In *You must have purchased this issue or have a qualifying subscription to access this content It has been identified that the institution you are trying to access this article from has institutional site license access to Scientific American on nature.com. Click here to access this article in its entirety through site license access. ADVERTISEMENT Scientific American is a trademark of Scientific American, Inc., used with permission © 2013 Scientific American, a Division of Nature America, Inc. All Rights Reserved.