08/14/2026
Could gene editing help treat inherited sight loss?
Andrew has lived all his life with Best disease, a rare inherited eye condition that affects central vision.
"If I look directly at your face, I can see your hair either side of it, but I can't see any features," he says. "That's why my eyes are always darting around, trying to pick up the detail."
Best disease is caused by a mutation in a gene called BEST1, and over time causes central vision to gradually deteriorate.
But a team of researchers at the London Project to Cure Blindness are hoping to change that.
Using skin cell donated by patients, they’re growing retinal cells in the lab that carry the same genetic mutation as the patient. Allowing them to study what goes wrong inside the cells in real time.
A big challenge is that there isn’t just one form of Best disease. Scientists have found more than 500 different disease-causing mutations.
So instead of developing a separate treatment for each one, the team asked: what if we could switch off the faulty copy of the gene while leaving the healthy copy untouched?
Using CRISPR gene-editing technology, that idea became possible with Andrews donated cells.
"It was really exciting," says Professor Amanda Carr. "We could see the cells recovering."
While the results are promising, researchers still need to develop safe and effective ways to deliver gene-editing therapies into patients' eyes before they can become treatments.
For Andrew, that progress represents something bigger than his own eyesight. He inherited Best disease from his father and has passed it on to his eldest daughter.
"I don't think the condition defines me," he says. "It's how I cope with it that defines me. If Amanda and her team managed to find a cure, I'd definitely take it."
Research like this is bringing hope to families affected by inherited sight loss and helping pave the way for future treatments.
Read the full story: https://www.ukri.org/news-and-events/lives-and-livelihoods/health/one-step-closer-to-curing-blindness/
