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.
For 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.
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.
"We're really excited about this," Dr. Eric Pierce, a professor of ophthalmology at Harvard Medical School, who is leading a study that the procedure launched, tells NPR.
"We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine," Pierce says.
