STAT+: A gene therapy designed to heal skin wounds helped preserve a boy’s vision
The case study, about how Krystal Biotech's gene therapy for a rare genetic condition also restored vision, was published in the NEJM.
In the clinical trial, the gene therapy seemed to be helping the boy’s skin wounds heal. But, his doctors wondered, could it also help his eyes?
The boy, Antonio Vento Carvajal, had a genetic condition called epidermolysis bullosa, or EB, which causes the skin to be so fragile that even the slightest friction can cause blisters or tears. Antonio, like some patients, also had eye issues as a result of the disease.
His eyelids were fusing to the eyes themselves. The eyes’ white tissue was growing over his corneas. The problems caused his vision to deteriorate to the point that, in his right eye, he could detect nothing more than the motion of someone waving a hand in front of his face.
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