Boy Gets New Skin, Thanks To Gene-corrected Stem Cells

The pink layer in the image is the extracellular matrix which helps the epidermis stay attached to the underlying

Hassan, a 7-year-old Syrian boy living in Germany, was born with a condition called epidermolysis bullosa that prevented the outer layer of skin from binding to the inner layer and left him with painful blisters starting when he was just a few days old, NPR reports. He was in a lot of pain, and doctors "had a lot of trouble keeping this kid alive", Tobias Rothoeft, a doctor at the hospital, said in a press briefing. Why do I have to live this life?

He's back to school, he's exercising, he's started to play's quite awesome. "I couldn't answer these questions". But because of the boy's poor prognosis, his parents gave their consent for expanding the experimental treatment on their son, and the authorities quickly granted approval on compassionate grounds.

"If he gets any bruises like small kids. have, they just heal as normal skin heals. like bruises in any other kids do".

"It was a tough decision for us, but we wanted to try for (our son)", the boy's father said.

The case study revolves around a boy with junctional epidermolysis bullosa, a disease that can take different forms depending on the specific genetic mutation. The result is skin that readily blisters, causing large, chronic wounds and vast pain to the patient.

To put in simple terms the therapy meant that a piece of his damaged skin was taken to the laboratory.

Afterwards, they could grow skin grafts containing this healthy gene, and applied it over the boy's body.

The boy was in the hospital for more than eight months, but now he is healthy.

So far, no problems have been detected. De Luca was able to grow entire sheets of engineered epidermis, which were then shipped to the hospital in Germany, NPR reported. He said, "Hassan feels like a normal person now, he plays, he's being active, he's enjoying his life and he's not the way he was before".

"After almost two months we were absolutely sure that we could do nothing for this kid and that he would die", Rothoeft told journalists ahead of the study's publication in the journal Nature. He was missing large areas of skin that made him exceptionally vulnerable to infections and other complications. Marinkovich said many patients don't survive beyond age 2 and that using the treatment for babies could be even riskier.

"[It] establishes a landmark in the field of stem cell therapy", Elaine Fuchs, a skin scientist at the Rockefeller University who also did not participate in the research, writes in an email to The Scientist.

In this photo taken on October 18, 2017, doctors lift up a sheet of skin in a lab at St Josef-Hospital in Bochum, Germany. After trying everything they knew, they concluded the child would die.

Since arriving in Germany with his family, the boy's condition had significantly deteriorated-De Luca suspects because of the family's distressing relocation, the resulting lack of consistent clinical care, and the family's language difficulties. He says the change has been "like a dream". During the many months since, he has not had so much as a blister, and loves to show off his "new skin".

This Associated Press series was produced in partnership with the Howard Hughes Medical Institute's Department of Science Education.



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