Collaborative Success Stories: Assalting Disease
University of Missouri scientists have uncovered clues about how drugs can rectify the harmful salt imbalance in cystic fibrosis.
Cystic fibrosis (CF) creates an imbalance of salt in the body, causing mucous to thicken in the lungs. Despite advances in treatments, the majority of individuals with CF die of respiratory failure. The illness is caused by mutations in a gene called CFTR, which encodes a homonymous protein that plays a key role in salt transport in the airway.
Now, a team of MU researchers led by physiologist Tzyh-Chang Hwang has identified two amino acids in the CFTR protein that act as a gate, regulating the flow of chloride ions in and out of cells through this important transport protein. Chloride is one of the key ingredients of salt.
Although there is currently no cure for CF, a drug is available that targets the defective CFTR protein. Until now it has not been clear precisely how the drug functions; the MU team’s discovery sheds light not only on how the drug works, but also on where it works. The findings could help researchers improve upon the drug’s effects and may also lay the foundation for the development of new medications.
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One Health/One Medicine