Drug discovery approach reveals why drugs for cystic fibrosis fall short
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16 June 2022Drug discovery approach reveals why drugs for cystic fibrosis fall short
To cure rare genetic diseases, from cystic fibrosis to Niemann-Pick, scientists at Scripps Research have turned to a computational approach usually used to pinpoint the best spot for an oil well. By using the method to analyze the spatial relationships between different variants of a protein—instead of the relationships between test wells across a landscape—the researchers can obtain valuable information on how disease affects a protein’s underlying shape and how drugs can restore that shape to normal.
