A new gene-editing technology called CRISPR is now being used to repair a genetic mutation that causes retinitis pigmentosa (RP), which is an inherited condition that degrades the retina and leads to blindness in at least 1.5 million cases around the globe.
This is the first time that researchers replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue. “Our vision is to develop a personalized approach to treating eye disease,” says Stephen Tsang, MD, PhD, the László Z. Bitó Associate Professor of Ophthalmology and associate professor of Pathology & Cell Biology at CUMC, ophthalmologist at New York-Presbyterian/Columbia, and one of the study’s senior authors. He adds that this is just the beginning, but that the results are promising: “We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible.”