Gene therapy partly restores sight in blind patients

Summarised from 2 outlets · Updated 8 Oct, 17:14 · Archive

Optogenetic gene therapy has partially restored vision in ten people with advanced retinitis pigmentosa.

Optogenetic therapy, based on Nobel prize-winning science for controlling nerve cells with light, has been shown to safely partly restore vision in blind people. The approach was first demonstrated in a single patient in 2021. Researchers have now reported results from a larger trial involving that original patient and nine others, all with advanced retinitis pigmentosa, a group of genetic disorders that affect light-sensitive cells in the retina.

The treatment involves a single injection into the eye. This injection contains a harmless, synthetic virus that delivers genetic instructions for producing a light-sensitive protein into a ring of surviving ganglion cells in the retina. These cells, which send visual information to the brain, deteriorate less quickly than the primary cells targeted by the disease, making them suitable candidates for the therapy.

Professor Botond Roska, director of the Institute of Molecular and Clinical Ophthalmology Basel and a lead author of the study, said: 'What we can say for sure is that the concept of optogenetics is valid for vision restoration.' Roska added that researchers now aim to work towards restoring high-resolution vision, with the hope of achieving this goal in five to ten years. Retinitis pigmentosa is thought to affect more than 1.5 million people worldwide.

How it is being reported

• A cure for blindness is comingA Nobel-winning therapy that can activate nerves with the flick of a switch is also being turned into treatments for deafness, pain and epilepsyThe i Paper · 8 Oct, 17:00
• Gene therapy can partly restore sight in blind people, researchers revealOptogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light A type of gene therapy based on Nobel prize-winning science is safe and can partly restore sight in blind people, researchers have revealed. The approach, known as optogenetic therapy, harnesses the technique for turning nerve cells on and off using light that scooped the 2026 Nobel prize in physiology or medicine. Continue reading...The Guardian · 7 Oct, 22:00

In this story: Institute of Molecular and Clinical Ophthalmology Basel · University of Pittsburgh Medical Center · Botond Roska · José-Alain Sahel

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