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During our 50th anniversary year, Retina UK was the focus of a BBC Lifeline Appeal on Sunday 16 November 2025.

Luxturna, a gene therapy, is only for the treatment of Leber congenital amaurosis type 2 (LCA2) and severe early-onset RP caused by mutations in a specific gene called RPE65.

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Biopharmaceutical company ProQR is conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat Leber's Congenital Amourosis (LCA) 10.

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• Leber congenital amaurosis (LCA)

Research determines that taking high dose vitamin A supplements does not slow vision loss in people with retinitis pigmentosa (RP).

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Our monthly e-Newsletter featuring the latest updates from Retina UK. Subscribe now to receive these updates directly to your email.

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Fancy fundraising for Retina UK? Here’s a few pointers to help get you started.

The following questions are often asked by people contacting Retina UK.

Elena Piotter, a PhD student working in Robert MacLaren’s group in the Nuffield Laboratory of Ophthalmology at the University of Oxford, is currently researching DNA and RNA base editing tools aiming to correct pathogenic mutations in ABCA4.

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Genes exist inside all the cells that make up your body. They contain genetic code (DNA) and provide instructions for constructing proteins, which are the building blocks of our bodies and perform a huge variety of roles.

Your doctor or genetic counsellor will spend some time asking about other people in your family to try to work out the way in which your particular faulty gene has been passed down through the generations – this is referred to as the inheritance pattern.