Columbia researchers have created a new technology using synthetic llama antibodies to prevent specific proteins from being destroyed inside cells. The approach could be used to treat dozens of diseases, including cystic fibrosis, that arise from the destruction of imperfect but still perfectly functional proteins.
In many genetic diseases, including cystic fibrosis, mutated proteins are capable of performing their jobs but are tagged for destruction by the cell’s quality control mechanisms.
Henry Colecraft (left) and Scott Kanner (photo by Diane Bondareff)
“The situation is analogous to ugly fruit,” says Henry Colecraft, PhD, the John C. Dalton Professor of Physiology & Cellular Biophysics, who led the research. “Shoppers reject fruit that doesn’t look perfect, even though ugly fruit is just as nutritious. If mutated proteins in cystic fibrosis can escape the cell’s quality control mechanisms, they work pretty well.”