One-Time Gene Therapy Restores Vision in 6-Year-Old with Rare Congenital Blindness
By
Andy Corbley
Summary
A 6-year-old girl named Saffie Sandford from Stevenage, UK, has had her vision saved through a one-time gene therapy for Leber's Congenital Amaurosis (LCA), a rare form of blindness caused by a mutation in the RPE 65 gene. The condition, which both her parents unknowingly carried a copy of, typically affects children and causes difficulty seeing in both low light and daylight. Thanks to the gene therapy treatment, Saffie can now see normally both day and night.
Source
bskyOne-Time Gene Therapy Restores Vision in 6-Year-Old with Rare Congenital Blindnessgoodnewsnetwork.orgKey quotes
· 3 pulledSaffie and her parents – credit, Great Ormond Street Hospital
Saffie Sandford from Stevenage was diagnosed with Leber's Congenital Amaurosis, (LCA) a mutation in the RPE 65 gene which both her parents unknowingly had a copy of.
LCA typically affects children and manifests as the inability to see in low light, and the difficulty to see in daylight.
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