Gene therapy restores some vision to blind patients
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Ten patients with retinitis pigmentosa in a clinical trial regained some light sensitivity after receiving a gene therapy that introduced a light-sensitive algae protein into their retinas.
Scientists at the Institute of Molecular and Clinical Ophthalmology Basel conducted the PIONEER study, combining gene therapy with optogenetics, a technique using light to control neurons. They injected a harmless virus carrying instructions for the algae protein, ChrimsonR, into the patients’ eyes.
The therapy aimed to make ganglion cells, which transmit signals from the retina to the brain, sensitive to light, as the patients' original light-sensing cells were failing. Participants then wore specialized goggles that scan the environment and deliver amber light pulses to activate the algae protein. Seven of the ten patients showed improved light sensitivity, and roughly half could then perform tasks like touching objects or identifying a black bar.
Improvements lasted for about five years in those who responded, and brain scans showed that the patients’ brains adapted to the new visual input. While the treatment does not restore full vision, sight remained blurry and limited, the study demonstrated the safety of the approach and suggests it could offer a potential treatment for various vision-related conditions. Researchers are now working on improving the goggles and the light-sensitive proteins to increase resolution and accuracy.
