A Gene-Editing Experiment Let These Patients With Vision Loss See Color Again
Researchers are working with a revolutionary gene-editing tool called CRISPR to treat patients with a rare eye disease. The seven patients have volunteered to allow doctors to modify their DNA by injecting the tool directly into cells still in their bodies. This marks the first time that researchers have used CRISPR this way, as earlier experiments had removed cells from patients’ bodies and later infused the modified cells back into the patients. On Wednesday, researchers revealed that they had found evidence that the approach appears to be working, as some patients witnessed improved vision. The seven patients suffer from a condition called Leber congenital amaurosis (LCA), which is a severe form of vision impairment.
More patients will need to be treated and tested to confirm if the approach is safe and to determine exactly how much it may help individuals with this condition. However, the results from the first trial are so promising that the researchers behind the trial have gotten approval to begin testing the treatment on the next group of patients. LCA is caused by a genetic mutation that disables crucial cells located in the retina. This results in patients experiencing a progressive loss of vision starting at birth that typically renders them legally blind at some point in their lives.