Six-Year-Old Saffie Sandford Regains Night Vision Through Luxturna Gene Therapy at GOSH

Saffie Sandford, from Stevenage, diagnosed at five with Leber's Congenital Amaurosis (LCA), a rare genetic disorder preventing the eye from producing a vital protein for normal vision, has undergone pioneering gene therapy. Her mother, Lisa, described the outcome as if "someone waved a magic wand and restored her sight in the dark."

Following tests at Moorfields Eye Hospital, Saffie received Luxturna therapy at Great Ormond Street Hospital (GOSH). This one-off treatment involves injecting a healthy gene copy directly into each eye. Without this intervention, Saffie faced complete blindness by the age of 30.

GOSH has identified this treatment as the first of its kind for one of the genetic causes of LCA. Saffie had procedures on one eye in April 2025 and the second in September 2025. Her mother noted that Saffie is now "thriving" and her peripheral daylight vision has also improved.

Scientists at GOSH and University College London, who tracked 15 children treated with Luxturna between 2020 and 2023, confirmed the therapy can enhance sight and strengthen visual pathways during crucial stages of brain development. However, they stressed it is not a complete cure. Younger children showed more significant improvements, indicating the importance of early intervention.

Dr. Rob Henderson, a consultant ophthalmologist at GOSH, highlighted that the research provides objective evidence of gene therapy's ability to bolster visual pathways in very young children with this condition. He emphasised that even minor improvements in vision can profoundly impact the lives of these children and their families, underscoring the therapy's potential and the need for age-appropriate outcome measures.