An avalanche of medical successes this year are sharing a common theme — genes. Gene editing is allowing researchers to more efficiently remedy conditions such as paralysis and leukemia. Though initially an unlikely candidate, gene therapy is now also instrumental in treating junctional epidermolysis bullosa. It saw its first triumph on a “butterfly boy” in Germany.
[Doctors] took a patch of non-blistered skin from the boy’s leg and used a virus to carry a corrected version of the bad DNA into his skin cells.
They grew grafts of the corrected skin and, in three separate operations over several months, replaced the missing skin.
Considering most “butterfly children” don’t make it past 30, genetic skin grafting could make an impact commercially. The therapy corrects stem cells, regenerating healthy substitutes. Since his discharge, the German schoolboy has remained healthy, living without the need for medication.
“This is really the way to go. You can get to the patients early before they have all the complications and suffering,”
With a growing population of “butterfly children”, this breakthrough could potentially relieve a giant itching epidemic.