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Scientists have taken another important step toward restoring vision in people with certain inherited forms of blindness. In a study published in the New England Journal of Medicine, researchers at the University of Pittsburgh School of Medicine and international collaborators showed that an experimental, optogenetics-based treatment was safely administered to 10 patients and improved visual function in some participants when used in combination with a specially designed visual stimulation device.

The retina, a thin layer of tissue at the back of the eye that captures light and sends visual information to the brain, is part of the central nervous system and thus, unlike many other tissues in the body, has a limited ability to repair itself.

Retinitis pigmentosa (RP), which destroys light-sensing cells in the retina, affects more than 1.5 million people worldwide. At onset, patients first notice impaired night vision, then impaired peripheral vision that gradually worsens over time and can ultimately lead to severe vision loss or blindness.

"Retinitis pigmentosa can result from a mutation in any one of more than 100 distinct genes, so it presents an extraordinary challenge," said José-Alain Sahel, distinguished professor and chair of the Department of Ophthalmology at Pitt, director of the UPMC Vision Institute and first and co-corresponding author of the study.

While in recent years Sahel and others have made strides in stopping and even reversing other progressive blinding diseases using gene therapies that target specific disease-causing mutations , RP requires an outside-the-box approach.

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