Gene therapy gives 'butterfly' boy Hassan new skin

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

Transgenic skin stem cells combined with improved tissue engineering methods have enabled an nearly total autologous skin transplant for a 7-year-old child with junctional epidermolysis bullosa (JEB), a rare genetic disease that causes the top layer of skin, the epidermis, to separate from the underlying dermis.

They grew sheets of skin and transplanted them onto the boy's body. No thanks to the genetic skin disease, about 80 percent of his epidermis had already been destroyed, with established treatments failing to give the child relief. The team took stem cells from an area of the patient's skin with no blisters and used this to grow new sections of epidermis that contained the non-mutated from of the gene, LAMB3.

Hassan, then seven years old, was admitted to hospital in June 2015 with the "devastating" effects of a genetic disease called Junctional epidermolysis bullosa (JEB) that has tormented him since birth.

As the discussion turned to making the boy's last days as comfortable as possible, his parents urged doctors to try "anything".

De Luca is a professor of biochemistry at the Italian University of Modena and Reggio Emilia's Center for Regenerative Medicine, and the senior author of the Nature paper reporting the case. "After two months we were absolutely sure we could do nothing for this kid and he would die".

In desperation doctors contacted experts in other countries, eventually getting in touch with Dr de Luca who was investigating experimental skin regeneration treatments. Remaining spots were covered in a later, smaller procedure.

That was two years ago, and today the boy is going to school, playing soccer and generally living the life of a normal child, the researchers say. "It doesn't blister at all and functionality is quite good".

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"If he gets any bruises like small kids. have, they just heal as normal skin heals. like bruises in any other kids do". It results in extremely fragile skin prone to breaking and tearing, and patients with such severe cases often don't survive beyond adolescence. This was the first time such an operation had been attempted on a large scale, and it raised questions as to whether the patient's body would accept the modified graft, and if the transplanted skin would continue to function properly.

The epidermis, tough and water-resistant, protects the body from dehydration, injury and infection. It's now the two-year anniversary of the procedure, and the boy is out of the hospital, off all pain medications, and regenerating healthy skin cells on his own.

Plastic surgeon Professor Tobias Hirsch said: "He'd lost almost two-thirds of his skin".

De Luca, from the University of Modena, said that the new epidermis "looks basically normal" and the patient is able to live a regular life.

The medical team obtained stem cells from Hassan, transferring an intact gene into the sample.

"Longer-term follow-up of the child in the current study and other patients will be needed to ensure that there are no adverse consequences - for example, the development of skin cancers", the duo wrote.

Even quite similar trials, though, have not seen comparable rates of insertional mutagenesis, and De Luca said that in skin transplants, "all the preclinical data we have point to a lack of toxicity", and in clinical work on skin grafts, clinicians have applied "on the order of" 100 million transduced cells, and never observed carcinoma.

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