Gene Therapy Restores Vision to Some of the Blind
What’s the Latest Development?
By injecting a virus with a normal gene directly into the retina of an eye with a defective gene, researchers have successfully restored some sight to more than a dozen people with a rare inherited eye disease called Leber’s congenital amaurosis, which leads to complete loss of vision in early adulthood. “They achieved this by inserting this corrective gene into adeno-associated viruses (AAV), and injecting these common but benign respiratory viruses directly into the retina. The photoreceptor cells take up the viruses and incorporate the functional gene into their nucleus to make a critical protein that the defective gene could not, rescuing the photoreceptors and restoring sight.”
What’s the Big Idea?
David Schaffer, professor of chemical and biomolecular engineering and director of the Berkeley Stem Cell Center at UC Berkeley, and who also led the experiment, said: “Building upon 14 years of research, we have now created a virus that you just inject into the liquid vitreous humor inside the eye, and it delivers genes to a very difficult-to-reach population of delicate retinal cells in a way that is surgically non-invasive and safe. It’s a 15-minute procedure, and you can likely go home that day.” With further research, Schaffer believes that the virus could knock out genes that are actively killing retina cells, which may be the case in age-related macular degeneration.
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