![]() Pierce, Albright and others stressed that only one patient has been treated so far and that the study, still at a very early stage, is designed primarily to determine whether injecting the gene-editing tool directly into the eye is safe. And it gives us hope that we could extend that to lots of other diseases - if it works and if it's safe." ![]() "This is the first time that's being tried in a human being. "All of us dream that a time might be coming where we could apply this approach for thousands of diseases," Collins tells NPR. "We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way," Albright says.įrancis Collins, director of the National Institutes of Health, calls the advance "a significant moment." Shots - Health News Molecular Scissors Could Help Keep Some Viral Illnesses At Bay "We're really optimistic that this has a good chance of being effective." "It's the first time the CRISPR gene-editing is used directly in a patient," Pierce says. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells - enabling patients to regain at least some vision. The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. "The majority of people affected by this disease have the most severe end of the spectrum, in terms of how poor their vision is," Pierce says. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement. In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery.īeginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. "We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine," Pierce tells NPR. Pierce is leading a study that the procedure launched. Eric Pierce, a professor of ophthalmology at Harvard Medical School and director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear. "We're really excited about this," says Dr. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored. The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see. KTSDesign/Science Photo Library/Getty Imagesįor the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body. Scientists at the Casey Eye Institute, in Portland, Ore., have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness.
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