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Second Dose of Gene Therapy for Inherited Blindness Proves Safe in Animal Studies
Date:3/3/2010

er 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.

In the current study, the study team found no evidence of toxic side effects in the blood or the eyes of the 10 animals—six dogs and four monkeys—that received the gene therapy. 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.

Researchers from The Children's Hospital of Philadelphia and from the University of Pennsylvania School of Medicine, and colleagues from two other institutions published their study today in the journal Science Translational Medicine. The first authors are Defne Amado, of the F.M. Kirby Center for Molecular Ophthalmology at Penn, and Federico Mingozzi, Ph.D., of the Center for Cellular and Molecular Therapeutics a
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SOURCE The Children's Hospital of Philadelphia
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