Improving the efficiency of cystic fibrosis airway gene therapy
Dr Martin Donnelley
Women’s and Children’s Health Network
Treatments for cystic fibrosis (CF) have improved in recent years, enhancing survival and quality of life, but these interventions are not curative. Gene therapy is likely the only mutation-class-independent method of overcoming CF lung disease, however achieving therapeutic levels of gene transduction is a challenge. This study will assess whether altering our current gene vector pseudotype ‘“ the coating that controls the types of cells transduced ‘“ can result in higher levels of airway gene expression. This will allow us to assess which pseudotype to progress towards clinical trials.