Researchers at Washington University School of Medicine in St. Louis have received a three-year $2.6 million grant from the Cystic Fibrosis Foundation to develop gene therapies for cystic fibrosis. In this disease, inherited mutations in a specific gene — called CFTR — cause thick mucus to build up in the lungs and impair breathing.
Led by David T. Curiel, MD, PhD, the Distinguished Professor of Radiation Oncology, and Ronald C. Rubenstein, MD, PhD, a professor of pediatrics and the Robert C. Strunk Chair for Lung and Respiratory Research in the Edward Mallinckrodt Department of Pediatrics, the grant will support development of a gene therapy that can insert the healthy gene into specific populations of a patients’ lung cells using a virus delivery system.
Past gene therapies for cystic fibrosis have not proven effective in large part because of how difficult it is to deliver a gene to the appropriate cell type in the airway using a viral vector. The lining of the lungs has many natural immune defenses against viruses, even when such therapeutic viruses are engineered to not cause disease. The funding will support their investigation into the innovative design of a viral vector that can effectively ferry into the lungs the healthy gene along with CRISPR/Cas9 gene-editing machinery. The goal of the strategy is to permanently insert the healthy gene into the appropriate lung cells.
