Cystic Fibrosis

About

Overview Cystic fibrosis (CF) is the most common life-threatening recessive genetic condition affecting Australian children. While there is no cure, the faulty gene responsible for the condition has been identified as the CFTR gene, and research now focuses on how to repair or replace it. Inventor Harsha Padmanabhan’s research aims to contribute to this goal by developing an aerosol spray for delivering the lentiviral vector carrying the correct CFTR gene. Background Gene therapy involves inserting a working copy of the faulty gene. The concept is to use a viral vector (lentivirus) to carry the CFTR copy, and insert it into the CF patient’s lung. Harsha’s research is looking at developing this gene therapy into an aerosol spray, which will enable it to be delivered non-invasively. This would be an incredibly useful application, as we head towards clinical trials of gene therapy for CF in the future.