Description
Luxturna is a gene therapy used to treat inherited retinal dystrophy caused by mutations in both copies of the RPE65 gene. It is designed to restore vision in patients with this rare genetic condition, which can lead to progressive vision loss and blindness. Luxturna works by delivering a normal copy of the RPE65 gene directly into retinal cells through a one-time injection beneath the retina. This enables the cells to produce the functional enzyme necessary for normal vision.
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