Second Dose of Gene Therapy for Inherited Blindness Proves Safe in Animal Studies

ScienceDaily (Mar. 4, 2010) — Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 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 now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection.

These new findings suggest that patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the other eye for Leber's congenital amaurosis (LCA), a retinal disease that progresses to total blindness by adulthood. Researchers had exercised caution by treating only one eye in the human trial.

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