The research team has been actively working to develop gene therapy vectors aimed at treating individuals with NF before the onset of symptoms, including tumour formation. This therapeutic approach could be likened to a "cancer vaccine" in certain respects – it seeks to target a patient’s genes to prevent future disease. 

This presentation will focus on the potential of CRISPR/Cas gene editing to correct underlying gene mutations causative for NF and adeno-associated viral vectors (AAV vectors) that will facilitate therapeutic delivery.