Translated material
Doctors restored vision to children using a new method of genetic therapy
The British doctors told The Guardian about partial vision restoration in four children with congenital genetic disease, in which patients can distinguish only light and darkness and completely lose their eyesight over time. After the operations that took place five years ago, the children have improved vision, and now they can distinguish objects, recognize the faces of parents, and some have learned to read and write.
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“It is difficult to overestimate the influence of even a slight improvement in vision. This is an opportunity to raise what he dropped, find a toy, it is better to navigate. I think, I think, as long -term advantages, with complete vision or without it, affect its development, ”commented the results of the treatment of the boy from the United States, who took part in the study.
All four children were diagnosed with Leber Amaurosis - an innate genetic disease in which retinal dystrophy occurs, leading to complete blindness. This condition causes a defect in the AIPL1 gene. In 2019-2020, the doctors introduced a healthy copy of this gene in one eye, the operation took only 60 minutes. All children received treatment from one to three years.
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Professor of ophthalmology Michel Michaelidis said that Amavrosis Leber is the most severe form of children's blindness. He noted that doctors first have the opportunity to effectively treat this disease.
The study published in the Lancet journal said that the treatment caused cystic macular edema in one of the patients who managed to cure. Doctors did not reveal other side effects.
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