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- W2051733086 abstract "Patients with cystic fibrosis commonly live into their teens, thanks to advances in disease management, but clinicians still cannot prescribe a treatment that addresses the underlying genetic problem. “Gene transfer has been tried and found to be inadequate so far for a variety of reasons,” said Richard Moss, director of the Cystic Fibrosis Center at Stanford University in California. “We also don’t have actual protein replacement at this point,” he said, referring to delivery of normal cystic fibrosis transmembrane conductance regulator (CFTR), a protein, to epithelial cells in the lungs and elsewhere. “We do have a series of drugs that affect both the mucus and ion transport” problems that result from insufficient amounts of normal CFTR, Moss added. And a variety of drugs are available to treat the airway inflammation, infection, and tissue destruction that eventually ensues. But under development, he said at an October 31 workshop in San Francisco sponsored by the Biotechnology Industry Organization, are “upstream” drugs that correct salt transport and cell function." @default.
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- W2051733086 date "2008-12-15" @default.
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- W2051733086 title "Drug developers aim to treat cystic fibrosis through disease modification" @default.
- W2051733086 doi "https://doi.org/10.2146/news080096" @default.
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