Life-changing treatment gives Saffie her sight back

Saffie, wearing purple glasses, holding a paint brush in one hand. She has on purple glasses and has long dark hair. She is sitting by a table in a kitchen, with cabinets all around her.

6 hours ago

Saffie has a rare condition called Leber's Congenital Amaurosis (LCA), which prevents cells in the eye from making a specific protein needed for normal vision.

But thanks to a new life-changing treatment she has now got her sight back.

The six-year-old's sight problems were inherited, which means they were connected to the genes passed from her parents.

But doctors were able to use gene therapy, an experimental medical technique that treats or prevents genetic diseases by inserting healthy genes into a patient's cells to replace or fix faulty genes.

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Saffie from Stevenage had worn glasses from the age of two and her mum Lisa, said the family was told, she would have been "blind by the age of 30" without treatment.

She had a procedure on one eye in April 2025 and the other in September, where a healthy copy of the gene was injected directly into each eye.

Lisa said now it's "like someone waved a magic wand and restored her sight in the dark".

After tests at Moorfields Eye Hospital in London she was treated at Great Ormond Street Hospital (GOSH), and received the Luxturna gene therapy.

Three people all together smiling and looking at the camera. The young girl in the middle is holding a toy and has on a pink and brown leopard print coat, with purple glasses on. A woman is to the left, with long dark hair and a man to the right with a black hat on, and black coat with a furry hood. — Image caption,
Saffie's mum Lisa said that the results of the treatment have been incredible

Before the treatment Saffie's mum Lisa said she struggled to see when there was low light, which meant having to use a torch to do everyday things like eating a meal, colouring, or going to a party.

But now, her mum says, she is able to do many more activities than she could before, including Trick or Treating with her family.

Saffie's peripheral sight - to see objects and movement outside of the direct line of vision - has also improved in the daylight.

A young girl, with her mouth open, looking excited, wearing clear glasses, and an Easter hat with chicks, flowers and bright colours on her head. She is by boxes in a room with a chair to the left.

Rob Henderson from GOSH, said: "For the first time, we've been able to show objectively that gene therapy can strengthen the visual pathways in babies and young children who are living with this rare eye condition."

Scientists at GOSH and University College London said that, while research showed the therapy can improve sight and strengthen visual pathways at a critical stage of brain development, it was not a cure.

More improvement has been seen in younger children with the treatment delivered during a critical period of visual development, compared to older children.