Sponging out cystic fibrosis

A Canadian team has found that compounds from sea sponges could be useful for treating cystic fibrosis. The compounds work in part by inhibiting a new family of targets for the disease: poly(ADP-ribose) polymerases.¹ The researchers now plan to work backward to uncover why blocking these polymerases improves the function of cystic fibrosis transmembrane conductance regulator, the mutated protein that causes cystic fibrosis.

CF results from loss-of-function mutations in cystic fibrosis transmembrane conductance regulator (CFTR), an ion channel that helps lubricate the epithelial lining of lungs, pancreas and intestine. Patients who inherit two mutated copies of CFTR develop thick mucus and are prone to severe respiratory infections and digestive problems...