The nine-year-old Syrian boy was dying from a severe condition called junctional epidermolysis bullosa, which caused him to lose the majority of his skin’s outer layer.
It left him with constant blisters and open sores and he was taken to hospital in June 2015, with doctors placing him in an induced coma to ease his suffering.
Doctors at the children’s hospital at Ruhr University in Bochum, Germany, tried using skin grafts from his father and donors, but they all failed.
The child was being prepared for palliative care, but after his desperate parents asked about experimental treatments, Dr Michele De Luca, of the University of Modena in Italy, was called in to help.
He and his colleagues had previously used gene therapy to produce a small piece of skin in a similar case.
The boy’s parents were told their son may not survive the complicated surgeries.”It was a tough decision for us. But we wanted to try for our son,” his father said.”We were forced to do something dramatic because this kid was dying,” said Dr De Luca.
Doctors took a small piece of the boy’s limited healthy skin and added those cells to the diseased gene to genetically modify them in a laboratory.
They then grew sheets of the modified skin, totalling a square metreIt took three operations for the doctors to transplant the lab-grown skin onto the boy, eventually covering 80% of his body.
Just 10 days later, his new skin was already beginning to grow, Dr De Luca said.
Eight months on, doctors say nearly all of the boy’s skin has been generated by the modified skin cells.
He is now able to play football, and does not even have to take medication.
Dr Tobias Rothoeft, one of the doctors originally looking after the boy, said: “This kid is back to his normal life again.”That’s what we dreamed of doing and it was possible.”
The science behind the case was published on Wednesday in the journal, Nature.”This takes us a huge step forward,” said Dr Peter Marinkovich, of Stanford University School of Medicine, who has carried out related work.
He said it was impressive the doctors were able to make such large amounts of viable skin after correcting the genetic defect.But he said in more serious cases complications, such as skin blistering in the lungs, the procedure may not work.He said many patients do not survive beyond age two and using the treatment for babies could be even riskier.
Dr Holm Schneider, of the University Hospital Erlangen in Germany, warned some severely ill patients might have an extreme reaction to skin transplants with an added gene.