(NPR) — For the first time, scientists have used a revolutionary gene-editing tool, CRISPR, to edit DNA while it's still inside a person's body.
They hope to restore vision to a patient blinded by a rare genetic disorder.
Doctors used CRISPR to edit genes of cells inside a patient's eye, hoping to restore vision to a person blinded by a rare genetic disorder. A similar strategy might work for some brain diseases.
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. 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 procedure, which takes about an hour to perform, involves making tiny incisions that enable access to the back of the eye. That allows a surgeon to inject three droplets of fluid containing billions of copies of the virus that has been engineered to carry the CRISPR gene-editing instructions under the retina.