What is ‘Leber congenital amaurosis’, recently seen in news?
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Researchers utilized CRISPR-Cas9 to restore vision in individuals with Leber congenital amaurosis (LCA), a rare genetic eye disorder causing blindness from birth. LCA affects 1 in 40,000 people, impairing light-sensitive cells due to a mutation in the CEP290 gene. The “BRILLIANCE” trial administered a single dose of CRISPR therapy, EDIT-101, which corrected the mutation, enabling proper function of the CEP290 protein, thus restoring vision by allowing the retina to perceive light.