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Millions of older Americans suffer from gradual vision loss as they reach their twilight years. But each year thousands of young people abruptly face a similar yet far more sudden fate… one that can leave grade- schoolers blind for the rest of their lives.
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One of the causes of hereditary blindness in boys and young men is called retinoschisis (reht-inn-oh-SKEE-siss), a disease that causes the retina — the light-processing structure in the back of the eye — to split in half, triggering rapid vision loss. Now University of Florida researchers, in collaboration with German and Canadian scientists, say they have cured the disease in male mice that had the same faulty gene that causes retinoschisis in humans. To stop the eye damage, the team used gene therapy techniques to transport into the eye a healthy version of the gene that keeps the retina intact.
Dr. William Hauswirth / UF Geneticist:
“So we’re simply putting in an extra copy, and this copy we’re putting in now is normal and it now makes a normal protein which is normally stable and does what it will do, and that is, get secreted from the cell and actually keep the retina, the other layers of the retina, from splitting."
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Although retinoschisis is just one of many genetic diseases that cause vision loss, scientists think this gene replacement method could be applied in some way to other eye disorders in the future. U-f researchers say the treatment prevented vision loss for the life of the study mice and may be tested in humans within two to five years. Doctors say the apparent beauty of this gene therapy method is in its simplicity.
Dr. William Hauswirth / UF Geneticist:
“The idea of the therapy is simple: If you’re missing a gene for a vital function, for vision, for seeing, the therapy is to replace that gene, put it back into the right cells in the retina so that those cells will function again. And that’s basically what we did.”
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From the University of Florida Health Science Center, I’m Eva Egensteiner.
