Correction of RPGR mutation in patient iPSC line.
CUMC and Uni of Iowa scientists have used 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. It 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.
“Our vision is to develop a personalized approach to treating eye disease,” says A/Prof Stephen Tsang, MD, PhD. “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...
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