“The CFTR protein acts like the airport gate at the top of the body’s epithelial cells—the cells lining the lungs, gut and pancreas,” she says. “When the gate opens, salts and water—the planes—flow out, to hydrate the mucus that traps bacteria in these organs and clear them out of the body.” But, with cystic fibrosis, the “airport” can not only malfunction—sometimes it doesn’t exist at all.

A/Prof. Waters’ lab has spent years developing more personalized ways of matching children to the right CFTR modulator drug. The lab method grows a patient’s own stem cells and turns them into tiny replicas of their lungs or gut.