A true medical revolution has taken place, with a 14-year-old American, Antonio Vento Carvajal, regaining his sight after years of blindness. Antonio, afflicted with a genetic disease called dystrophic epidermolysis bullosa, benefited from a clinical trial of the world’s first topical gene therapy.
The initial therapy, a gel, significantly improved Antonio’s skin condition, prompting Dr. Alfonso Sabater to suggest that the same treatment might also help to remove the scars on Antonio’s eyes, impeding his vision. After consulting with Krystal Biotech, the manufacturer of the treatment, a formula adapted for ocular use was developed.
This new treatment, Vyjuvek, uses an inactivated herpes simplex virus to introduce functional copies of the affected gene. After extensive testing and approval for compassionate use by the United States Food and Drug Administration, Antonio received Vyjuvek as eye drops, administered with a syringe.
Antonio’s vision gradually improved, eventually reaching a clarity of 20/25 in one eye and 20/50 in the other. Today, with his sight restored, Antonio can finally do something he couldn’t do before: play video games with his friends.
This case marks an unprecedented milestone in the medical field, opening up incredible possibilities for the future of modern medicine thanks to the advent of topical gene therapy. It is concrete proof of the power of gene therapy and its potential to revolutionize the treatment of genetic diseases.