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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.

Researchers develop gene therapy for rare ciliopathy

Researchers 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.

Gene Therapy for Rare Ciliopathy Developed

Researchers have developed a gene therapy that rescues cilia defects to treat a rare disease that causes blindness in early childhood.

NIH researchers develop gene therapy for rare ciliopathy

NIH researchers develop gene therapy for rare ciliopathy
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