Science: helping the blind see.
Scientists at Columbia University Medical Center (CUMC) and the University of Iowa, in a combined effort, have used gene-editing technology called CRISPR to effectively repair a genetic mutation that causes retinitis pigmentosa (RP). The inherent genetic mutation causes a condition where the retina in the eye degrades completely, eventually leading to blindness. More than 1.5 million cases of this condition have been diagnosed around the world.
This has been the first successful attempt at replacing the defective gene associated with RP. The key to the success was CRISPR-Cas9 gene editing technology, a technique developed in 2013 which has since been the center of several controversies.
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The researchers, who published their results in Scientific Reports, took a sample of stem cells from the skin of a patient with retinitis pigmentosa. These derived stem cells contained the defective mutation for RP which the scientists replaced with a good copy of the same gene.
Because stem cells have the remarkable ability to transform and differentiate into different subtypes of cells, the scientists were able to transform these newly edited stem cells from the patient into healthy retinal cells, which were then transplanted back into the patient to restore lost vision.
The video below describes the process behind treating the RP sensory genetic disease: