Scientists at NIH’s National Eye Institute have developed a promising gene therapy strategy for a form of Leber congenital amaurosis (LCA), a rare disease that causes severe vision loss in childhood. The scientists tested their approach using lab-made retinal tissues built from patient cells, called retinal organoids, one of which is pictured here. ⠀

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Credit: Anand Swaroop, Ph.D., NEI/NIH

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