United States: Thanks to new therapy, a 14-year-old blind man has regained his sight

Delphine Schiltz // Photo credit: Ferdy Damman / ANP / AFP 07:24, July 27, 2023

Drops to regain sight. A priori, it seems very simple. Yet this is the feat that has just been achieved by a research team from the University of Miami, in the United States. The patient, Antonio Vento Carvajal, a 14-year-old boy blind due to a genetic disease, underwent topical gene therapy, a first in the world. It could pave the way for the treatment of many patients like him.

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An innovation for experts

The teenager suffered blisters and scars all over his body, including in his eyes. As a result, his cornea was blocked, which prevented him from seeing. In question, a genetic disease. He first received gene therapy treatment that introduces healthy genes into dysfunctional cells. Treatment in the form of a gel, first applied to the patient's skin, worked very well.

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So much so that this formula has been adapted into a drop for the eyes. A real innovation according to some experts. "First, this is a pretty devastating disease for which there has been no other treatment until now. And secondly, by creating a fairly unique product that is quite suitable for ophthalmological application, this new way of preparing drops, it can be toxic to a cornea that is already at the base very fragile, to an eye that is excessively fragile. So I think it's something innovative," says Isabelle Audo, professor of ophthalmology at the Vision Institute.

This is the very first application of a successful gout gene therapy. A great advance for all genetic diseases of the cornea.