Word: cystic
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...discovery has a multitude of practical implications. Cystic fibrosis, for example, and some forms of kidney disease are caused by the failure of key proteins to get where they ought to be. Understanding the details of such failures could probably lead to powerful treatments. Indeed, Blobel's research has already helped scientists use tiny cellular "factories" to mass-produce proteins such as erythropoietin, which stimulates red-blood-cell production. A deeper understanding of cellular machinery, which Blobel continues to pursue, could eventually show how cells are damaged in Alzheimer's disease, cancer and infections...
What makes the conversation tricky is that we're already on the slippery slope. Doctors can screen fetuses for genetic diseases like cystic fibrosis and Duchenne muscular dystrophy; one day they may be able to treat them in utero. But correcting is one thing, perfecting is another. If doctors can someday tinker with a gene to help children with autism, what's to prevent them from tinkering with other genes to make "normal" children smarter? Technology always adapts to demand; prenatal sex-selection tests designed to weed out inherited diseases that strike one gender or the other--hemophilia, for instance...
Entrepreneur O'Donnell is a former assistant dean at the business School and currently serves as chair of Boston Concessions Group. But he is better known in Boston as founder of the Joey Fund, a non-profit organization named for his late son and dedicated to helping those with cystic fibrosis...
Wilmut said positive aspects of cloning could include creating pigs with organs more acceptable to human bodies for transplants and using sheep to study cystic fibrosis...
...early gene-therapy trial for cystic fibrosis, inflammation caused by the viral carrier, an altered adenovirus, was so severe that the FDA ordered a halt to the effort, casting a pall over all the other trials--and the field in general. More problems plagued the researchers. In many cases the implanted genes failed to "turn on," or express themselves, and were unable to command the cells to produce the protein they were supposed to provide. Some operated for a while and then inexplicably shut down...