Boy with rare disease gets brand new skin with gene therapy

Boy with rare disease gets brand new skin with gene therapy

Boy with rare disease gets brand new skin with gene therapy

His new skin no longer blistered and was able to heal normally.

In a groundbreaking development, surgeons have regenerated the epidermis - the uppermost layer of the skin - of a 7-year-old child who was on the verge of death as a result of a lethal skin disease. The boy lost 80% of his skin and was battling multiple infections due to junctional epidermolysis bullosa (JEB), a rare genetic disorder which causes the epidermis of the skin to disconnect from the dermis, leading to severe wounds, infections (frequently fatal), and (oftentimes) cancer.

All forms of standard treatment proved hopeless, and the child's body rejected grafts taken from his father. Genetically modified stem cells were then cultivated and converted into transgenic transplants. Because known therapies were not able to help in managing Hassan's condition, it was then that the medical team chose to give an experimental approach a shot: transplanting skin developed from genetically modified stem cells over wound surfaces.

A period of four months inpatient recovery then followed, and Hassan was discharged in February 2016, the team said: "Transplanting 80% of the skin and providing intensive medical care to the patient over a period of eight months was extremely challenging". Today, almost two years since he got the transplant, Hassan has stress-resistant skin and is back to attending school and taking part in social activities with his family.

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Dr Michele de Luca, from the University of Modena, Italy, who led the gene therapy team, said: "The patient was in danger of life".

The skin grafts stand up to physical stress, and function in every way like skin should, without the need for ointments or other treatments, the researchers say in research published Wednesday in Nature.

"If we think about the experience we have in the burns, I would say that this epidermis would stay basically forever", he says. "It doesn't blister at all and functionality is quite good".

The health and longevity of the skin comes from the presence of certain long-lived stem cells in the epidermis.

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Grown in the laboratory, the repaired cells produced colonies of regenerative "mother" stem cells.

Observing these stem cells at work also helped clear up a mystery about how the skin functions. With the researchers new insights it's clear that the latter hypothesis is correct.

The "experimental" gene therapy was the last resort after all other options had failed for Hassan, who suffers from epidermolysis bullosa, otherwise known as a "butterfly child" because of the fragile and delicate nature of their skin.

The research could be applied to other JEB patients and other skin conditions, although the transplants may be more fraught with difficulty in adult patients, since their skin won't replenish itself monthly as easily as a child's will. In any case, the work is heartening both for the near total success of the technique in this case and for the affirmation that genetically engineered cells can integrate well with our own tissues even at large scales.

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