Boy with rare condition gets new skin with gene therapy
A 7-year-old boy with a rare genetic skin condition was saved by doctors who grew a replacement skin for him using gene therapy.
MODENA, ITALY — A young patient who lost most of his skin to a genetic disease now has a new chance at life, thanks to a breakthrough therapy that gave him replacement skin.
The New York Times reports that the 7-year-old boy was born with junctional epidermolysis bullosa, a condition in which a gene defect causes the skin to become fragile, often tearing and forming blisters.
By June 2015, the boy gotten two bacterial infections that destroyed two-thirds of his skin. He was treated with antibiotics, bandages, and even a skin graft from his father, but nothing worked.
Doctors decided to use gene therapy on a patch of non blistered skin, and used a virus to carry a corrected version of the defective gene into the skin cells.
The engineered cells were grown into sheets of skin and grafted onto the child's body to replace his missing skin.
The grafts took and grew, and the patient was discharged by February 2016. His epidermis is currently stable and doesn't blister or itch.
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