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1 shot of gene therapy and children with congenital blindness can now see
Date:10/24/2009

common in LCA. After treatment, seven of them had significant improvements in nystagmus.

Some of the most dramatic results, captured on video by the researchers, are apparent as subjects traverse a standardized obstacle course. Before the treatment, the patients had great difficulty avoiding barriers, especially in dim light. After treatment, the children navigated the course more quickly, with fewer errors than before, even at the lowest light levels. Not all the adults performed better on the mobility course, and for those who did, the improvements were more modest compared to the children's.

"In follow-up studies, we will continue to monitor these patients to determine whether this treatment stops the progression of this retinal degeneration," said Maguire. "In the future, we hope to investigate whether other retinal disease will be amenable to this gene therapy approach."


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Contact: Karen Kreeger
karen.kreeger@uphs.upenn.edu
215-459-0544
University of Pennsylvania School of Medicine
Source:Eurekalert  

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