Scientists save child's life by growing him new skin

Scientists save child's life by growing him new skin

His skin also appeared to have normal elasticity and wound-healing behavior. The boy, who has a genetic skin disease called junctional epidermolysis bullosa (JEB), had been expected to die. But his body rejected this. Each month, human skin is entirely replaced with new cells, but whether this renewal is the result of a large population of equally potent progenitor cells or of a smaller number of individual stem cells that dominate regeneration has been hotly debated. But after almost a decade of filing paperwork and improving his skin cell production system, De Luca was ready to try. Those patches were grafted onto the child's body over several months that brought him back from a near-death condition to a normal life.

The tissue was then grown into grafts that were used to replace 80 per cent of the patient's skin in three operations.

The boy, then 7, was hospitalized in June 2015 with blisters on his limbs, back and elsewhere. If even one of them can find relief using De Luca's technique - or a less intense version - it'll be worth the time and effort. In January 2016 the boy, whose name is not being released to protect his privacy, received a few more skin patches-and in February he was released from the University Hospitals of the Ruhr University Bochum in Germany. "The boy was so happy with his new skin that he wanted to show off".

The story of the child told in journal Nature and reported by Wired shows the enormous potential and promise of stem cell and gene therapies.

"Both theories were valid, but what had been lacking was real proof in vivo, in patients", says Aiuti.

Put simply, "it's a lovely piece of work", stem cell and regenerative medicine researcher Fiona Watt of King's College London writes to The Scientist about the team's accomplishment.

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He said the child was using a home trainer and playing football.

Some unusual pigmentation remains on the boy's skin because of the earlier damage. "He has never developed blisters where we transplanted him". "Now, he's a robust kid". People with the disease lack critical proteins that attach the outer layer of the skin to the inner layer, resulting in fragile skin with nearly constant blisters and open sores. The underlying dermis layer, he says, is so compromised that it's not clear it would be able to regrow epidermis. The boy has no scarring, because his disease was literally only skin-deep. However, the non-mutated LAMB3 genes in the transgenic skin cells that cover the boy's body seem to be enough to make them functionally healthy. He said it was impressive that De Luca and colleagues were able to make such large amounts of viable skin after correcting the genetic defect. The illness can be caused by one of several mutations.

A team of biologists who specialize in gene therapy came in from University of Modena and Reggio Emilia in Italy, and the boy's parents agreed to an experimental therapy. In his own research Marinkovich has used gene therapy to treat four patients with a different genetic form of epidermolysis bullosa.

Treating the boy had provided useful scientific information that improved understanding of how skin was regenerated and maintained, said the scientists. Before undergoing the experimental treatment, the young patient described in the study had lost much of his skin to the disease and was admitted to the hospital. The complicated treatment will not be cheap. Caring for children with epidermolysis bullosa is not cheap either, Marinkovich notes, adding that it can cost $100,000 a year to dress their wounds-which is sometimes not covered by insurance. The same group of scientists previously performed a similar procedure on two children with the same condition, but this is the first time they did so on someone with such a severe case. Prevention, De Luca says, is better than restoration.

Furthermore, it's unclear how long the boy's skin will remain healthy, meaning he'll need to go through additional examinations as he gets older. But no one really knows, because the approach is so new.

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