New gene therapy approach could help prevent blindness in children with Leber congenital amaurosis

Researchers from the National Eye Institute have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood.

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Kamil Kruczek ,Anand Swaroop ,Emily Welby ,Emily Henderson ,National Eye Institute ,National Institutes Of Health ,Clinical Center ,National Institutes ,Gene ,Gene Therapy ,Blindness ,Fell ,Ilia ,Na ,Eye ,Laboratory ,Leber Congenital Amaurosis ,Medical Research ,Rganoids ,Mh ,Protein ,Research ,