A recent gene therapy trial showed significant improvement in the vision of individuals with a rare genetic condition causing sight loss early in childhood. Patients experienced up to a 10,000-fold enhancement in their vision after receiving the therapy. The trial included 15 participants with the condition, Leber congenital amaurosis, resulting from mutations in the GUCY2D gene. The therapy involved injecting the gene therapy, ATSN-101, under the retina. The treatment was well-tolerated, with minor side effects related to the surgical procedure. The successful trial provides hope for treating congenital retinal blindness and suggests potential future treatments for other genetic retinal diseases.
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100-fold improvement in sight seen after gene therapy trial
