Washington, Apr 29 (UNI) Patients virtually blind have a reason to rejoice.
Researchers used gene therapy to increase light sensitivity and improve vision in patients who were virtually blind, a finding that offers new hope to hundreds of thousands of patients with inherited forms of vision impairment.
Although the patients had a rare form of blindness called Leber's congenital amaurosis, researchers believe the approach can ultimately be used for a broad spectrum of disorders, including retinitis pigmentosa and macular degeneration.
Surgery yields success The experiments, so far meant only to prove the safety of the technique, produced "real clinical benefit" and "made a real difference in patients' lives," said geneticist Robin R. Ali of University College London.
"The fact that they had patients who could now read lines on an eye chart . . . and one who could run an obstacle course -- this is a really great advance," said geneticist Stephen Rose, chief research officer of the Foundation Fighting Blindness, who was not involved in the research. "This has changed the landscape of hope for patients." According to Dr Morton F. Goldberg, an ophthalmologist at John Hopkins University's Wilmer Eye Institute: "In the field of retinal dystrophies, this is, I believe, the most important therapeutic discovery" in four decades. "It's a landmark." The results are particularly important because gene therapy, in which a defective gene is replaced with a good one, has been "a snakebitten field," said Dr Albert M Maguire of the University of Pennsylvania School of Medicine.
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