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.
Drug 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.