The trial named 'BRILLIANCE' focused on treating those with Leber Congenital Amaurosis (LCA), restoring sight to participants with severe vision loss from a gene mutation impairing the CEP290 protein essential for vision. The CRISPR-Cas9 gene therapy, EDIT-101, precisely targeted and corrected the mutation, allowing the retina to function properly. This groundbreaking approach restored normal vision for those affected.
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