A 7-year-old who lost most of his skin to a rare genetic disease has made a dramatic recovery after receiving an experimental gene therapy, researchers announced today. The treatment—a whole-body graft of genetically modified stem cells—is the most ambitious attempt yet to treat a severe form of epidermolysis bullosa (EB), an often-fatal group of conditions that cause skin to blister and tear off at the slightest touch.
The new approach can address only a subset of the genetic mutations that cause EB. But the boy’s impressive recovery—he’s now back in school and is even playing soccer—could yield insights that help researchers use stem cells to treat other genetic skin conditions.
“It is very unusual that we would see a publication with a single case study anymore, but this one is a little different,” says Jakub Tolar, a bone marrow transplant physician at the Masonic Cancer Center, University of Minnesota in Minneapolis who is developing …
READ MORE ON SCIENCEMAG.ORG