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Gene delivery may reverse childhood blindness
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GAINESVILLE, Fla. — Scientists have been successful here in delivering a gene through an eggshell to restore sight to a chicken that would otherwise be born blind, the University of Florida said in a statement. A similar treatment should be able to be developed to eradicate Leber congenital amaurosis type 1 in children as well, the researchers said.
“We were able to restore function to the photoreceptor cells in the retinas of an avian model of a disease that is one of the more common causes of inherited blindness in human infants,” Sue Semple-Rowland, PhD, said in the statement.
Of the seven treated chickens, five displayed “near-normal” visual behavior, the researchers said. Tests on three untreated chickens showed no meaningful responses, they said.
Children with LCA1 would receive an injection of the gene transfer agent directly into the eyeball during the first few years of life, the researchers said.