Gene Therapy Restores Vision

(Ivanhoe Newswire) -- Scientists have discovered a new technique to treat serious genetic optical diseases, giving patients hope for a clearer future.

Researchers at the University of Pennsylvania School of Medicine used gene therapy to treat 12 patients with Leber congenital amaurosis (LCA), a serious and rare disease of the eye caused by at least one of 13 possible genetic mutations. All 12 patients' vision was improved from the gene therapy and increased at least 100-fold in constricting of the pupil when exposed to light.

In gene therapy, doctors insert genes directly into a patient’s cells and tissue. In this study, a virus carrying the genetic mutation was injected into the eye with the worst vision. The 12 patients were ages eight to 44 and suffered from LCA.

“All 12 patients given gene therapy in one eye showed improvement in retinal function,” study authors wrote. “The effect was stable during follow-up. The results support our hypothesis that the response to subretinal gene therapy depends on the extent of retinal degeneration and, therefore, the age of the patient.”

The researchers say their success in treating this rare disease provides the foundation to treating other retinal diseases.

LCA causes patients to begin losing their vision at a rapid rate as an infant or early in childhood and also causes abnormal eye movements. Total blindness is common by the 30s and 40s. The condition is considered very rare, affecting about five babies annually in the United States.

SOURCE: The Lancet, 2009

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