Gene therapy gives 'butterfly' boy Hassan new skin

Gene therapy gives 'butterfly' boy Hassan new skin

This procedure's success demonstrates that the combination therapy may be effective against some rare genetic skin disorders.

Details of the treatment, previously only used to reconstruct small areas of skin in two patients, appear in the latest issue of Nature journal.

The boy's disease was caused by a mutation in a gene, called LAMB3, that produces a protein that anchors the epidermis to the deeper layers of skin beneath. Children with the condition have mutations in one of three genes - LAMA3, LAMB3 or LAMC2. Genetically modified stem cells were then cultivated and converted into transgenic transplants.

A boy who almost died when disease stripped most of the skin from his body, is playing soccer two years after he received a new, gene-edited hide in an experimental procedure, the doctors who treated him said. More than 40 percent die before adolescence.

Researchers Tobias Rothoeft and Tobias Hirsch said: "The close collaboration between the departments in Bochum and the University of Modena's expertise have been the key to success".

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

"Once you have regenerated the epidermis, the stem cells keep making the renewal of the epidermis as in a normal [healthy person]", said De Luca. De Luca and colleagues had pioneered techniques correcting the same genetic defect. This leaves his skin as fragile as butterfly wings, vulnerable to blistering and erosion. But the boy, who has a mutation in the LAMB3 gene, had lost almost all the skin on his back and legs and had blistering in other areas.

In September 2015, the team took a 4-square-centimeter patch of unblistered skin from the boy's groin and grew skin stem cells in the lab from that sample.

They tried to promote "spontaneous healing" with strong antibiotics, which did not work, then transplanted skin from Hassan's boy's father, which was rejected.

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After permissions were obtained for the compassionate use of an experimental therapy, the skin was grafted onto the boy's body in two operations in October and November 2015.

De Luca and a team took skin cells from an unaffected part of the boy's body, fixed the mutated gene, and used the corrected cells to grow skin in culture.

Grown in the laboratory, the repaired cells produced colonies of regenerative "mother" stem cells. Sheets of lab-grown cells were transplanted to the boy.

The approach was successful thanks to the structure of the skin itself.

Two years later, the doctors report the boy is doing so well that he doesn't need any medication, is back in school and even playing soccer. "He plays soccer", says Hirsch. He still has some blistering in untreated areas, and his doctors are considering replacing more skin. "To replace and gene-correct the whole skin of a patient is just wonderful".

To try and save him from that, scientists took a tiny sample of skin from the child. Still, he said the approach was worth trying in dying patients. "He's not a mouse". But a new therapy changed all that.

Treating Hassan has provided useful scientific information that improved understanding of how skin was regenerated and maintained, said the scientists.

The health and longevity of the skin comes from the presence of certain long-lived stem cells in the epidermis. Each stem cell can then either copy itself or morph into a variety of different types of mature skin cells.

Microscope images show that in normal skin (left) the top layer, called the epidermis (purple in bottom images), is fused to the dermis by laminin 332 (green, in top images) and other proteins. Jouni Uitto, the chairman of the department of dermatology and cutaneous biology at the Sidney Kimmel Medical College at Thomas Jefferson University in Philadelphia also warns that epidermolysis bullosa sometimes affects internal structures, such as the lining of the esophagus and urinary tract, and gene therapy for skin can not address these problems. He quickly lost about 60 percent of the outer layer of his skin and was put into an induced coma to spare him further suffering.

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