Gene therapy provides the opportunity to cure all those affected by cystic fibrosis (CF), but this approach has been met with several challenges preventing its clinical success. The goal of this workshop is to address the current barriers to effective treatment of CF using gene therapy and introduce methods to overcome them using state-of-the art genetic and cell-based therapeutic approaches for CF.
Describe therapeutic approaches with the potential to provide long-lasting benefit in the treatment of cystic fibrosis.
Identify the challenges remaining in achieving an effective one-time CF therapy using genetic and cell-based approaches.
Explain the latest advances in genetic and cell-based approaches in the treatment of CF.
M.A., M.D., FRCP, F.Med.Sci.,
Professor of Gene Therapy & Respiratory Medicine/Honorary Consultant Physician,
National Heart & Lung Institute