Penn Medicine - March 3, 2010 - Second Dose of Gene Therapy for Inherited Blindness Proves Safe in Animal Studies

PHILADELPHIA – 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.

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