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<meta name="description" content="Columbia University Medical Center (CUMC) and University of Iowa scientists have used a new gene-editing technology called CRISPR to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition." />
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CRISPR Used to Repair Blindness-causing Genetic Defect in Patient-derived Stem Cells
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A potential precision medicine approach for treating vision loss
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January 27, 2016
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<a href="/about/news-and-events/news?topic=401" hreflang="en">Gene Therapy</a>
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<a href="/about/news-and-events/news?topic=56" hreflang="en">Rare Diseases</a>
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<p>Columbia University Medical Center (CUMC) and University of Iowa scientists have used a new gene-editing technology called CRISPR to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide.</p><p>The study marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue.</p>
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