A boy suffering from ‘epidermolysis bullosa’ got treated successfully with Gene Therapy

Epidermolysis bullosa, a kind of genetic skin disease, generally affects the outer layer of the skin. A boy suffering from this disease got treated by using transplants taken from genetically modified cells. This treatment has become successful to heal this skin disease. This is a great achievement for scientists. 

Image credit Epidermolysis Bullosa

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This disease had damaged 80% outer layer of the skin of the boy and all types of possible treatments could not cure the disease. University of modena located in Italy and a medical team from at the Ruhr-Universitat Bochum established in Germany conducted a trial which transplanted the skin of the boy taken from genetically modified stem cells to put on damaged surface of the skin.

2 years have gone when the boy was treated. Now he is normal and spending good social life. 

Do you know epidermolysis bullosa? 

It is a congenital skin disease and called epidermolysis bullosa as the scientific name. Currently there is no such treatment which can cure this disease.

For skin regeneration our body needs protein-forming genes. When the production of protein forming genes reduces it affects the skin regeneration process. In this condition a small stress causes skin damage and starts to form scar tissue. This disease may also damage the internal organs of our body and also it can be a life threatening issue.

Hassan was 7 years old when he was taken to the hospital named Katholisches Klinikum Bochum and admitted to paediatric ICU department. Till then 60% of his skin was destroyed. This incident happened in june 2015.

"He suffered from severe sepsis with high fever, and his body weight had dropped to a mere 17 kilogrammes - a life- threatening condition," said Tobias Rothoeft, Consultant at the University Children's Hospital at Katholisches Klinikum Bochum.

To see his skin condition doctors decided to transplant his skin with genetically modified epidermal stem cells. Doctors managed to get these cells from the patient through skin biopsy process and transplanted all the damaged skin tissues happened in the boy's body. There were several places on the body that were destroyed due to epidermolysis bullosa.

"Overall, 0.94 square metres of transgenic epidermis were transplanted onto the young patient in order to cover all defects, accounting for 80 per cent of his entire body surface," said Tobias Hirsch, head consultant at the department of plastic surgery.

It was the first transplantation held in October 2015 which developed the patient's skin condition. The transplanted stem cells successfully created the new skin.

In February last year, the patient was discharged. Today, almost two years after the experimental therapy was initiated, high-quality, stress-resistant skin with intact hydrolipid film, as well as early formation of hair, researchers said.

No scar contractures have appeared in transplanted areas. Hassan is attending school again and is actively taking part in his family's social life, they said.

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