World first gene therapy trial for thalassemia at Royal Prince Alfred Hospital

A world first gene therapy trial is currently underway at Royal Prince Alfred Hospital with the potential to cure patients living with thalassemia. Patient Stacey Wong has required long frequent hospital visits for most of her 26 years but that may be all behind her. RPA’s head of molecular therapies, Professor John Rasko, said the trial was “just the beginning of what we hope is going to be a revolution, not just in thalassemia, or just blood disease, but in many different forms of genetic abnormality. The sky is the limit”.