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Viewpoint: Experimental gene therapy saved my sons’ eyesight

| | December 6, 2019
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Anthony and his brother Nicholas, both who have LCA. Image: Kristin Papiro
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Not long after we brought my newborn son, Anthony, home from the hospital, we noticed his eyes kept darting to the nearest light. If left in a room alone, he couldn’t self-soothe unless we placed him beside a sunlit window out of which he would obsessively gaze. He was eventually diagnosed with Leber congenital amaurosis, or LCA, a rare retinal disease affecting one out of every 50,000 newborns.

For Anthony, it was like viewing the world permanently through a pair of sunglasses. It was only a matter of time, the doctors agreed, before the lights went out completely.

At age 6, Anthony became one of the first patients in human clinical trials for Spark Therapeutics’s experimental gene therapy to treat this rare form of congenital blindness.

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After the treatment Anthony had fresh observations about my eye color and wedding pictures that had been on our walls for years. Follow-up eye exams measuring visual acuity and light sensitivity confirmed what was plainly obvious: Gene therapy hadn’t only halted Anthony’s vision loss; it had yielded measurable vision gain. The permanent sunglasses weren’t off completely, but they were several shades brighter.

Read full, original post: Genomic Science Kept My Boys from Going Blind

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