Are UC Berkeley scientists on the verge of a breakthrough that could help cure blindness?
A recently discovered but yet relatively simple form of gene therapy was able to restore sight in blind mice within a month of treatment, researchers with the university announced Friday in a news release.
The treated mice were “navigating around obstacles as easily as mice with no vision problems,” the release said.
Scientists at UC Berkeley say the method could have the potential to restore treatment in blind humans who have lost sight due to retinal degeneration, restoring a sufficient amount of vision to let them navigate or even read.
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“With neurodegenerative diseases of the retina, often all people try to do is halt or slow further degeneration,” molecular and cell biology professor Ehud Isacoff said in a statement. “But something that restores an image in a few months – it is an amazing thing to think about.”
The therapy could theoretically be delivered by an injection of an inactivated virus into an eyeball.
As UC Berkeley’s news release explains, retinal degeneration is a complex problem with hundreds of different mutations possible. However, some layers of retinal cells, including subretinal ganglion cells, can remain healthy long after blindness takes hold, even though these cells are not sensitive to light.
Other studies in recent years, including a 2017 report by Oxford scientists, have shown that restorative gene therapy delivered via subretinal injections is possible in humans.
In trials, the research team at UC Berkeley inserted green-light receptors into the eyes of blind mice, finding that 90 percent of ganglion cells gained light sensitivity with the treatment.
The technology involved in the newly found process has been around for a while, scientists said.
“That this system works is really, really satisfying, in part because it’s also very simple,” Isacoff said in Friday’s release. “Ironically, you could have done this 20 years ago.”
The research was published online Friday in the scientific journal Nature Communications. It was co-authored by Isacoff, fellow molecular biology professor John Flannery and their colleagues. They had been researching the topic for more than a decade, according to UC Berkeley’s news release.
Isacoff and Flannery are raising funds, hoping to begin human trials for the gene therapy in the U.S. in the next three years.
“When everyone says it will never work and that you’re crazy, usually that means you are onto something,” Flannery said.