A remarkable new treatment not only restores blind individuals’ vision, but it has also been found to strengthen visual pathways in the brain. In clinical trials, “gene therapy” was used to treat patients afflicted with a rare inherited disease (Leber’s congenital amaurosis Type 2), which causes their retinas to degenerate slowly. These patients typically have limited vision at birth, and then progressively lose their remaining vision by midlife. Each patient underwent gene therapy in only one eye, which involved injecting the retinas with a harmless virus that inserts good copies of genes that are defective in the individual. The study participants not only regained their vision, but the unused visual pathways in their brains were as healthy as sighted individuals.
Clinical trials are underway that will help determine if gene therapy can be used more widely as a treatment for blindness.
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