Oct 25, 2009 8:53 am US/Pacific
Vision Restoration Breakthrough Injects Genes
PHILADELPHIA (CBS) ―
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A study is using gene therapy to safely improve vision in five children and seven adults with Leber's congenital Amaurosis (LCA). The greatest improvements occurred in the children. Currently, there is no treatment for LCA. (File)
CBS
Born with a rare retinal disease that made him legally blind, Corey Haas, used to ride his bike just in front of his house with help from others. The 8-year-old boy can now ride around his neighborhood all by himself.
Corey told CBS station KYW-TV, "In school, I can recognize people by their faces not just their voices."
His vision has improved after a single injection of genes that produce light-sensitive pigments in the back of his eye. His treatment represents the next step toward medical science's goal of using gene therapy to cure Leber's congenital Amaurosis (LCA).
Corey's father, Ethan, added, "I'm so happy for him and it's like a dream come true."
The study, conducted by researchers from the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia and the Department of Ophthalmology at the University of Pennsylvania School of Medicine, used gene therapy to safely improve vision in five children and seven adults with LCA. The greatest improvements occurred in the children. Currently, there is no treatment for LCA.
"To do something like this, it's a little bit like trying to land a man on the moon. You're under tremendous pressure to get it right the first time," Katherine High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia said.
Jean Bennett, M.D., Ph.D., F.M. Kirby professor of Ophthalmology at the University of Pennsylvania School of Medicine explained, "In the United States, there are about an estimated two thousand people with LCA."
Although the patients did not attain normal eyesight, 6 out of 12 patients participating in the clinical trial improved enough that they may no longer be classified as legally blind. The clinical benefits have persisted for almost two years since the first subjects were treated with injections of therapeutic genes into their retinas.
Bennett added, "The individual who's two years out now is continuing to gain vision, not as quickly as she did in the beginning but it hasn't decreased."
In follow-up studies, researchers plan to continue to monitor the patients to determine whether this treatment stops the progression of the retinal degeneration.
RELATED LINKS:
Children With Congenital Blindness Can See After Gene Therapy
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