Cystic Fibrosis Foundation supports new gene therapy collaboration with UC Berkeley and the Innovative Genomics Institute

Most patients with cystic fibrosis (CF) can now be treated with combinations of drugs that help the mutant CFTR protein reach the cell surface and function more normally. However, many less common CFTR mutations cause forms of CF that cannot be treated using currently available drugs. Advances in CRISPR technology open new possibilities for correct the CFTR gene using a targeted enzyme that is capable of finding the region containing the genetic mistake and reversing that genetic error. This is the focus of a new CF Foundation-supported Collaborative Research Program based at UC Berkeley and Innovative Genomics Institute (led by Fyodor Urnov, Jennifer Doudna, and Ross Wilson), UCSF (the Erle lab and longtime collaborator Professor Walt Finkbeiner), and UAB (Steve Rowe). At UCSF, we will be testing new delivery systems developed at IGI in cultured primary human airway epithelial cell models.