A study of Editas Medicine's CRISPR-based therapy to treat Leber congenital amaurosis has begun, NPR reports.
It adds that the study is the first to rely on CRISPR editing within the human body. Other CRISPR-based approaches typically remove patients' cells, edit them, and then return the modified version. But NPR notes that as Leber congenital amaurosis affect fragile retinal cells, the researchers instead decided to use modified viruses to deliver the CRISPR machinery to the eye to make the changes in the body.
"This is the very first time that anyone's ever actually tried to do gene-editing from inside the body," Lisa Michaels, chief medical officer at Editas, tells NPR. "We're actually delivering the gene-editing apparatus to the part of the body where the disease takes place in order to correct it."
As NPR reports, the treatment targets the CEP290 gene, one cause of Leber congenital amaurosis. So far, researchers have treated four people and hope to treat six adults and, eventually, eight children, it adds. The hope is to prevent further vision loss and possibly restore some vision that has already been lost, it notes.