For the first time, scientists have used CRISPR to edit DNA while it's still inside a person's body, NPR has learned.
"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.
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. Still, he says, if the underlying defect can be repaired in this patient and others with advanced damage,"we have the potential to restore vision to people who never had normal vision before. It would indeed be amazing."If the approach appears to be safe and effective, the researchers will start treating younger patients.
The idea is that once there, the CRISPR editing elements would snip out the mutation that causes a defect in CEP290. The hope is that this would be a one-time treatment that would correct vision for a lifetime.
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