A new study involving the revolutionary gene-editing technique called CRISPR, which allows scientists to make precise changes in DNA, is designed to try to restore vision to patients who suffer from a rare genetic disease National Public Radio reports.
Doctors think CRISPR could help patients fighting many diseases, including blood disorders such as sickle cell disease and several forms of cancer
But in those experiments, NPR’s Rob Stein reports, doctors take cells out of the body, edit them in the lab and then infuse the genetically edited cells back into patients.
This new experiment marks the first time scientists are using CRISPR to edit DNA when it’s still inside patients’ bodies.
“We’re optimistic,” said Dr. Eric Pierce of Mass Eye and Ear, a member of Mass General Brigham in Boston, where the first patients were treated.
They were born with a form of Leber congenital amaurosis, which destroys light-sensing cells in the retina, destroying vision.