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Can Gene Therapy Cure the Blind?

By Melissa Lafsky
Apr 28, 2008 10:41 PMNov 5, 2019 8:40 AM


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The New England Journal of Medicine has published a report stating that scientists in the U.S. and the U.K. have performed the first successful use of gene therapy—the process of replacing "defective" genes with normal ones—to "dramatically improve" the sight of people with a rare hereditary eye disease that causes blindness. Six subjects received the treatment and four saw their vision partially restored, according to the AP, and experts predict that the technique, if it continues its success in more trials, could be used to treat other blindness-causing diseases. Testing only one eye in each subject (in case something went horribly wrong, which hasn't been an unheard-of occurrence in gene therapy testing), the researchers injected millions of copies of a normal, working gene under the retina. Following the treatment, each subject was given routine tests for light sensitivity and motility. Three of the subjects saw "significant" improvement, while the fourth reported only a minor change—though, admittedly, one that "makes a big difference" in his ability to function in the dark. The study represents good news for a field of research that has, to date, sucked up million dollars of funding over the past 15 years, with very little (thus far, anyway) actual results to show for it. Still, far be it from us to say gene therapy can't ever lead to successful methods of treating innumerable diseases. Plus anything that helps the blind see can't be all bad, in our book.

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