Gene-based light therapy restores limited sight in small trial of inherited blindness
علاج بصري وراثي يعيد قدرات إبصار محدودة لمصابين بعمى وراثي

A small clinical trial has produced encouraging results for a form of inherited blindness, with several patients regaining the ability to detect objects and pinpoint where they are, according to a study published in the New England Journal of Medicine.
The trial involved 10 patients who had lost their sight to retinitis pigmentosa, a genetic disease that destroys the eye's light-sensitive cells and can lead to severe vision loss. Seven of the 10 became more sensitive to light after treatment, and six showed clinically meaningful gains, including spotting objects in front of them, locating them and reaching for them more accurately.
The approach, known as optogenetic therapy, combines genetic modification of the retina with specially designed goggles. Doctors inject one eye with material carrying the genetic code for a light-sensitive protein called ChrimsonR, developed by GenSight Biologics, so that the retina's surviving nerve cells can respond to light. The goggles then capture visual information from the surroundings and convert it into light patterns at specific wavelengths, activating the protein and allowing visual signals to reach the brain.
The researchers stressed that the results do not amount to restored normal vision, but suggest some visual function can be recovered even in advanced blindness. They reported one serious medical event immediately after an injection, which resolved within minutes, and said larger studies are needed to assess long-term effectiveness and safety.
"Even in people with severe vision loss, the visual system retains a remarkable capacity to process new information," said lead author Dr. José-Alain Sahel of the University of Pittsburgh.


