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With single gene insertion, blind mice regain sight


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2019 Mar 16, 7:24am   292 views  0 comments

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It was surprisingly simple. University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems. They were able to see motion, brightness changes over a thousandfold range and fine detail on an iPad sufficient to distinguish letters.

The researchers say that, within as little as three years, the gene therapy — delivered via an inactivated virus — could be tried in humans who’ve lost sight because of retinal degeneration, ideally giving them enough vision to move around and potentially restoring their ability to read or watch video.

You would inject this virus into a person’s eye and, a couple months later, they’d be seeing something,” said Ehud Isacoff, a UC Berkeley professor of molecular and cell biology and director of the Helen Wills Neuroscience Institute. “With neurodegenerative diseases of the retina, often all people try to do is halt or slow further degeneration. But something that restores an image in a few months — it is an amazing thing to think about.”

About 170 million people worldwide live with age-related macular degeneration, which strikes one in 10 people over the age of 55, while 1.7 million people worldwide have the most common form of inherited blindness, retinitis pigmentosa, which typically leaves people blind by the age of 40.

“I have friends with no light perception, and their lifestyle is heart-wrenching,” said John Flannery, a UC Berkeley professor of molecular and cell biology who is on the School of Optometry faculty. “They have to consider what sighted people take for granted. For example, every time they go to a hotel, each room layout is a little different, and they need somebody to walk them around the room while they build a 3D map in their head. Everyday objects, like a low coffee table, can be a falling hazard. The burden of disease is enormous among people with severe, disabling vision loss, and they may be the first candidates for this kind of therapy.”

Currently, options for such patients are limited to an electronic eye implant hooked to a video camera that sits on a pair of glasses — an awkward, invasive and expensive setup that produces an image on the retina that is equivalent, currently, to a few hundred pixels. Normal, sharp vision involves millions of pixels.



The new therapy involves injecting inactivated viruses into the vitreous to carry a gene directly into ganglion cells. Earlier versions of viral therapy required injecting the viruses underneath the retina (bottom). The gene makes normally ‘blind’ ganglion cells light sensitive, returning sight to eyes that have lost the normal light sensors, the rods and cones. The right image shows the cell layers in a normal retina. (Image by John Flannery)

More: https://news.berkeley.edu/2019/03/15/with-single-gene-insertion-blind-mice-regain-sight/

Also at with links to related articles: https://www.sciencedaily.com/releases/2019/03/190315095808.htm

#Vision #Eyesight #Blindness #SciTech #Medicine
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