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Gene Therapy Partially Restores Sight in Blind Patients, Study Finds

A new gene therapy, optogenetic therapy, has been shown to be safe and can partially restore sight in people with advanced retinitis pigmentosa.

  • Optogenetic therapy uses a single eye injection and light-stimulating goggles to make retinal cells light-sensitive.
  • The therapy was tested on 10 participants with advanced retinitis pigmentosa, with 6 showing meaningful improvements in light sensitivity.
  • The technique does not depend on the exact genetic cause of sight loss and has shown stable restoration of some visual function for up to five years.

Researchers have revealed that a type of gene therapy, known as optogenetic therapy, is safe and can partly restore sight in blind people. This approach harnesses a technique for turning nerve cells on and off using light, which was awarded the 2026 Nobel Prize in physiology or medicine.

The therapy was initially shown to restore vision in one blind person in 2021. Now, a larger trial involving the original patient and nine additional individuals has reported results. All 10 participants had advanced retinitis pigmentosa, a group of genetic disorders affecting over 1.5 million people globally.

The gene therapy involves a single injection into the eye, delivering genetic instructions to make surviving ganglion cells light-sensitive. Patients then use light-stimulating goggles that convert images into light pulses, activating these cells and allowing the wearer to perceive a monochrome image.

The study, published in the New England Journal of Medicine, followed participants for up to five years. Six of the 10 participants experienced clinically meaningful improvements in light sensitivity. Some also showed improvements in tasks like detecting and locating objects while wearing the goggles.

Researchers noted that the study has limitations, as patients can detect objects but not yet faces. The team aims to work towards restoring high-resolution vision within five to 10 years.

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