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

January 27, 2016

Gene Therapy Genetics Rare Diseases Retinitis Pigmentosa Stem Cells

Translational Research

Grantee

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.

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.