STAT+: Gene therapy eyedrops restored a boy’s sight. Similar treatments could help millions
STAT
JULY 24, 2023
But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?
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