Scientists at Case Western Reserve University have developed gene-carrying nanoparticles they believe hold promise in treating Leber congenital amaurosis (LCA) and other congenital visual disorders.
Researchers reportedly focused on the form of the disease called Leber congenital amaurosis 2 (LCA2), where patients carry a mutated RPE65 gene and suffer from profound vision loss from birth. The researchers reportedly used a mouse model of the human form of LCA to test a lipid-based nanoparticle called ECO they designed to deliver healthy RPE65 genes to retinal pigment epithelium (RPE) cells. Following injection into the retina of mice, testing reportedly showed a significant increase in light-induced electrical activity from the eyes to the brain, indicating the rods and cones were operating as they should in the visual cycle.
Although this research focused on LCA2, the scientists reportedly believe their technology holds promise for other forms of LCA, as well as other inherited diseases that lead to severe vision loss or blindness.
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Source: Case Western Reserve University