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Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.
Antonio, who’s been legally blind for much of his 14 years, can see again.
The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?
This insight not only helped Antonio, it also opened the door to similar therapies that could potentially treat millions of people with other eye diseases, including common ones.
Antonio’s mom, Yunielkys “Yuni” Carvajal, teared up thinking about what Sabater did for her son.
“He’s been there through everything,” she said in Spanish during a visit to the University of Miami Health System’s Bascom Palmer Eye Institute. “He’s not only a good doctor but such a good human being and provided us with hope. He never gave up.”
The family came to the U.S. from Cuba in 2012 on a special visa allowing Antonio to get treatment for his condition, which affects around 3,000 people worldwide. He had surgeries to remove scar tissue from his eyes, but it grew back. Antonio’s vision kept getting worse, eventually deteriorating so much that he didn’t feel safe walking around.
Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions
Antonio Vento Carvajal has been legally blind for much of his 14 years. A gene therapy delivered through eyedrops is allowing him to see again and has opened the door to similar therapies that could potentially treat millions of people with other eye diseases.
Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.
Antonio, who’s been legally blind for much of his 14 years, can see again.
The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?
This insight not only helped Antonio, it also opened the door to similar therapies that could potentially treat millions of people with other eye diseases, including common ones.
Antonio’s mom, Yunielkys “Yuni” Carvajal, teared up thinking about what Sabater did for her son.
“He’s been there through everything,” she said in Spanish during a visit to the University of Miami Health System’s Bascom Palmer Eye Institute. “He’s not only a good doctor but such a good human being and provided us with hope. He never gave up.”
The family came to the U.S. from Cuba in 2012 on a special visa allowing Antonio to get treatment for his condition, which affects around 3,000 people worldwide. He had surgeries to remove scar tissue from his eyes, but it grew back. Antonio’s vision kept getting worse, eventually deteriorating so much that he didn’t feel safe walking around.
