Gene Therapy, Stem Cells: Prime for Vision Restoration

Editor's Note: The second installment of this five-part series, on hearing, will appear in the Oct. 1 issue. Using gene therapy, scientists earlier this year reversed blindness in three dogs afflicted with Leber congenital amaurosis (LCA). The news excited the scientific world and popular press. LCA is a rare, inherited disease characterized by a severe loss of vision at birth. Researchers at the University of Pennsylvania, Cornell University, and University of Florida showed that injecting a

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This research characterizes the current state of scientific investigation into the world of blindness: virtually all human trials are far down the road, and any findings, so far, pertain only to the particular disease itself. A bright side does exist: vision restoration is a prime candidate for both gene therapy and stem cell transplants. And other researchers are using different approaches in which electrodes implanted on the retina allow blind people to see edges and shapes thanks to electrode stimulation from a camera mounted on a pair of glasses. 2 These researchers, from Johns Hopkins University, are awaiting Food and Drug Administration approval for a Phase I trial, says project director Mark Humayun. "Even restoring some vision in blind patients can have a significant impact on their lives by restoring their independent mobility," Humayun says.

The American Foundation for the Blind estimates that about 10 million people in the United ...

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