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Sick boy gets healthy new skin with genetically-altered stem cells

13 November 2017
Appeared in BioNews 926

A boy with a rare skin disease has been successfully treated by replacing most of his skin with grafts of stem cells modified by gene therapy.

The Seven year old patient had severe epidermis bullosa (EB) which made his skin brittle, blistered, and prone to life-threatening infections. A team led by Professor Michele de Luca at the University of Modena and Reggio Emilia in Italy, treated the boy and reported their study in Nature.

'This is one of these [studies] that can determine where the future of the field is going to go,' said Dr Jakub Tolar, at the University of Minnesota in Minneapolis, to Science.

The boy's condition was due to a mutation in the laminin beta 3 gene (LAMB3). The researchers took a biopsy from an unblistered area of the patient's skin, and grew it over scaffolds in the laboratory to generate sheets of replacement skin: a well-known technique often used to treat burns patients. In this case however, retroviruses were used to insert a healthy copy of the LAMB3 gene into the cells before growing the sheets and grafting them to the patient's limbs, flanks and back.

This successfully replace 80 percent of the patient's skin, and after almost two years, the skin in the grafted areas remains strong and elastic. 'He hasn't developed a single blister,' Professor de Luca told The Atlantic. 'He's gaining weight. He's playing sports. He's got a normal social life.'

In the report, the researchers note that although attempts have been made to treat EB in similar ways, these treated such small areas that the patients' quality of life were not improved to the same extent.

Although the researchers noted that the same approach would not be possible with all EB patients, they hold that there are insights to be had for the treatment of similar conditions.

For example, they saw that the replacement skin was maintained by the presence of pools of long-lived and successfully repaired stem cells called holoclones. These holoclones can reproduce themselves and also produce progenitor cells which generate mature skin cells.

'Hence, the essential feature of any cultured epithelial graft is the presence (and preservation) of an adequate number of holoclone-forming cells,' wrote the authors.

A boy with a rare disease gets new skin, thanks to gene-corrected stem cells
Science |  8 November 2017
A Dying Boy Gets a New, Gene-Corrected Skin
The Atlantic |  8 November 2017
Gene Therapy Creates Replacement Skin to Save a Dying Boy
New York Times |  8 November 2017
Regeneration of the entire human epidermis using transgenic stem cells
Nature |  8 November 2017
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