Medical researchers have saved the life of a seven-year-old boy by growing genetically-modified replacement skin for him. The young German boy suffered from a deadly congenital condition called epidermolysis bullosa, a condition that cases the sufferer’s skin to tear and blister, as if it had been burned. The procedure not only saved his life, but he’s now able to participate in sports with his classmates.
By 2015 the patient had been admitted to the burns unit at Bochum Children’s Hospital in Germany: at that point, two-thirds of his skin was either was either badly damaged or outright missing, and traditional treatments failed to yield results, including skin grafts from a donor.
Scientists from the University of Modena in Italy were called in, to try an experimental skin regeneration technique they had been developing: using a sample of the lad’s epidermis, they removed the defective part of his genetic code that causes epidermolysis bullosa, and used the repaired genes to grow sheets of replacement skin using colonies of stem cells.
21 months after performing a series of surgeries to graft the lab-grown skin onto the young patient, the researchers report that the boy is not only happy and healthy, his newfound skin is robust enough for him to rough around with his schoolmates, including playing football.
"He went back to normal life, including school and sports," explains Dr Michele de Luca, the head of the gene therapy team. "His epidermis is stable; robust. It doesn’t blister at all and functionality is quite good."
Invaluable information regarding the skin’s regenerative capabilities was also gathered while the researchers were treating the patient. They found that "transgenic epidermal stem cells can regenerate a fully functional epidermis virtually indistinguishable from a normal epidermis," according to their study paper published in the journal Nature.
"The successful outcome of this study paves the way for gene therapy to treat other types of epidermolysis bullosa and provides a blueprint that can be applied to other stem cell-mediated ex-vivo (outside the body) cell and gene therapies."