A 6-year-old girl from Stevenage, UK has regained her vision thanks to a one-time gene therapy for Leber's Congenital Amaurosis (LCA), a rare form of blindness caused by a mutation in the RPE65 gene.
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6 Year Old Saffie Has Her Vision Saved from Rare Form of Blindness Thanks to One-Time Gene Therapy
6 Years Old Patient age
Leber'S Congenital Amaurosis (LCA) Condition
One-Time Gene Therapy Treatment type
RPE65 Gene affected
Full Day And Night Sight vision restored