Gene therapy for a severe inherited blindness disorder (Leber's congenital amaurosis or LCA), which produced dramatic improvements last year in twelve children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial has now reported that a study in large animals has shown that a second injection of the normal gene (RPE65) into the opposite, previously untreated, eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection. LCA is an autosomal recessive retinal disease that progresses to total blindness by adulthood and that can be caused by mutation in any one of a variety of different genes, including the RPE65 gene. Approximately 3,000 people in the United States are estimated to have the disease. In the current study, the research team found no evidence of toxic side effects in the blood or the eyes of the ten animals—six dogs and four monkeys—that received the gene therapy (with the normal RPE65 gene). Each animal received an injection first in the right eye, then in the left eye 14 days later. All six dogs, which had been specially bred to have congenital blindness, had improved vision, in addition to showing no toxic effects from the gene therapy. The monkeys, which were not blind, also showed no toxic effects from the therapy. These new findings suggest that LCA patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the other eye. Researchers had exercised caution by treating only one eye in the human trial. "We designed this study to investigate the immunological consequences of administering the gene therapy injection to the second eye after treating the first one," said senior author Dr.
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