The only treatment that was available was to carry out a cell bone marrow transplant from another individual in order to rebuild her white cells.
“But that meant a 30 per cent chance she would die, and her father did not want his child to take that risk. So he asked if he could enrol her in the gene therapy that we wanted to do.”
After treating the gene in her own bone marrow cells, she grew into a healthy child and was cured of her illness.
“This is all we had to give her – she did not have any other treatment,” he said.
He showed a recent picture of her, looking happy and healthy with her father.
“Her father often sends me pictures of her skiing and wearing ballet costumes to show how she is leading a normal life,” he said.
“This [gene therapy] will change the word of medicine because it will allow us to treat those severe genetic disorders.