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

Jennifer Fisher Wilson
Sep 16, 2001
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 good copy of RPE65 protein into the right eye of LCA-afflicted dogs restored sight in that eye.1 But human clinical trials are more than a year away.

Graphic: Lisa Damiani

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...