Combination of stem cells and gene therapy essentially cures a genetic skin disease
In a last-ditch effort to save a dying 7-year-old boy, scientists have used stem cells and gene therapy to replace about 80 percent of his skin.
This procedure’s success demonstrates that the combination therapy may be effective against some rare genetic skin disorders. The study also sheds light on how the skin replenishes itself, researchers report November 8 in Nature.
In 2015, a boy with a rare genetic skin condition, called junctional epidermolysis bullosa, had lost most of his skin and was close to death. Children with the condition have mutations in one of three genes — LAMA3, LAMB3 or LAMC2. Those genes produce parts of the laminin 332 protein, which helps attach the top layer of skin, the epidermis, to deeper layers.