Oct 23, 2020

Scientists use gene therapy and a novel light-sensing protein to restore vision in mice

NIH-funded therapy will now be tested in humans.

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for the development of MCO1. The company is planning a U.S. clinical trial for later this year.

Nanoscope’s findings, reported today in Nature Gene Therapy, show that totally blind mice—meaning they have no light perception—regain significant retinal function and vision after treatment. Studies described in the report showed that treated mice were significantly faster in standardized visual tests, such as navigating mazes and detecting changes in motion.

Opsins are proteins that signal other cells as part of a cascade of signals essential to visual perception. In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina. When activated by light, the photoreceptors pulse and send a signal through other retinal neurons, the optic nerve, and on to neurons in the brain.

A variety of common eye diseases, including age-related macular degeneration and retinitis pigmentosa, damage the photoreceptors, impairing vision. But while the photoreceptors may no longer fully function, other retinal neurons, including a class of cells called bipolar cells, remain intact. The investigators identified a way for bipolar cells to take on some of the work of damaged photoreceptors.

Illustration of eye cross sectionThe detail shows the structure of the retina, including the location of a bipolar cell expressing Nanoscope’s MCO1 opsin.

“The beauty of our strategy is its simplicity,” said Samarendra Mohanty, Ph.D., Nanoscope founder and corresponding author of a report on the mouse study that appears today in Nature Gene Therapy. “Bipolar cells are downstream from the photoreceptors, so when the MCO1 opsin gene is added to bipolar cells in a retina with nonfunctioning photoreceptors, light sensitivity is restored.”…..

Read more: https://www.nih.gov/news-events/news-releases/scientists-use-gene-therapy-novel-light-sensing-protein-restore-vision-mice

Source: National Institutes of Health

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