Principal investigator Eric Pierce pointed out that the trial shows that gene therapy for hereditary vision loss is a worthy pursuit for future research. He believes the early research is promising.
“It’s a big deal to hear how thrilled they were to finally be able to see food on their plates,” said Pierce. “These were individuals who couldn’t read a single line on an eye chart. They had no treatment options, which is an unfortunate reality for most people with inherited retinal disorders.”
The goal is to inject CRISPR so that it reaches the retina to restore the ability to produce genes and proteins.
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