Gene therapy can partly restore sight in blind people, researchers reveal
Source: Guardian Health ↗
A gene‑therapy technique called optogenetic therapy, which uses light to switch nerve cells on and off, has been shown to be safe and to partially restore vision in people with advanced retinitis pigmentosa. The study, led by Prof Botond Roska of the Institute of Molecular and Clinical Ophthalmology Basel and Dr José‑Alain Sahel of the University of Pittsburgh Medical Center, treated ten participants—one who had previously been the sole subject in a 2021 trial and nine new patients—by injecting a harmless synthetic virus into the eye. The virus delivers instructions for a light‑sensitive protein to surviving retinal ganglion cells, making them responsive to light. After the injection, each patient wears a pair of goggles that convert visual scenes into monochrome light pulses of a single wavelength, which activate the engineered ganglion cells and allow the wearer to perceive basic shapes and movements. The trial followed the treated eyes for up to five years, recording only one brief, severe eye‑related side effect that resolved within minutes and no systemic adverse events.
Six of the ten participants experienced clinically meaningful gains in light sensitivity, and several demonstrated functional improvements in tasks such as locating and touching a notebook, finding a door, and walking along a line while using the goggles. The researchers noted that participants who devoted more time to training with the device tended to achieve better performance, underscoring the importance of rehabilitation alongside the biological intervention. Because the therapy targets ganglion cells, which are less affected by the genetic mutations that cause retinitis pigmentosa, it works regardless of the specific genetic cause of the disease, which affects over 1.5 million people worldwide. However, the current approach yields only monochrome vision and cannot yet resolve faces, a limitation attributed to the treated ganglion cells being arranged in a ring around the fovea, the retinal region responsible for sharp central vision.
External experts praised the findings as a crucial safety validation and a step toward more functional vision restoration. Mark Hankins, a visual neuroscience professor at the University of Oxford who was not involved in the study, highlighted that the results demonstrate stable, multi‑year improvements in light sensitivity for patients who still retain some residual vision. The investigators aim to refine the method to achieve higher‑resolution, color vision within the next five to ten years, suggesting that optogenetic therapy could eventually become a broadly applicable treatment for a range of inherited retinal disorders.
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