Gene therapy helps treat life-threatening skin disease

Gene therapy helps treat life-threatening skin disease thumbnail
BERLIN: In a first, scientists have successfully treated a child suffering from extensive skin damage due to a genetic disease using transplants derived from genetically modified cells.

The boy suffers from epidermolysis bullosa, a genetic skin disease that had destroyed about 80 per cent of the outermost layer of his skin.

After all established therapies had failed, the medical team from at the Ruhr-Universitat Bochum in Germany and University of Modena in Italy decided to try an experimental approach.

They transplanted skin derived from genetically modified stem cells onto the wound surfaces.

Thanks to the successful therapy, the boy is now – two years after the treatment – able to participate in his family’s life and social life.

Epidermolysis bullosa is the scientific name of a congenital skin disease that is currently considered to be incurable.

Its underlying mechanism is a defect in protein-forming genes that are essential for skin regeneration. Even minor stress can result in blisters, wounds, and skin loss with scar formation.



Keep Reading This Article

Related posts