Cystic fibrosis (CF) is caused by a mutation of the CFTR gene. While there are many hundreds of known mutations, not all of them are currently treatable which means a significant number of CF patients lack targeted therapies. In an effort to identify new treatments for these patients, researchers from Boston University School of Medicine (BUSM) set out to use blood cells from individuals with CF to make patient-specific induced pluripotent stem cells (iPSCs) and generate lung epithelial cells in the lab. These lung cells are functional and highly similar to the lung cells of the patient. Using these ‘lung cells in a dish,’ they have created a novel platform to discover effective drugs for those patients who currently don’t have any treatment options.
