Scientists say they have used the gene editing tool CRISPR inside someone’s body for the first time
The people in this study have Leber congenital amaurosis, caused by a gene mutation that keeps the body from making a protein needed to convert light into signals to the brain, which enables sight. They’re often born with little vision and can lose even that within a few years.
Scientists can’t treat it with standard gene therapy -- supplying a replacement gene -- because the one needed is too big to fit inside the disabled viruses that are used to ferry it into cells.
So they’re aiming to edit, or delete the mutation by making two cuts on either side of it. The hope is that the ends of DNA will reconnect and allow the gene to work as it should.
It’s done in an hour-long surgery under general anesthesia. Through a tube the width of a hair, doctors drip three drops of fluid containing the gene editing machinery just beneath the retina, the lining at the back of the eye that contains the light-sensing cells.
“Once the cell is edited, it’s permanent and that cell will persist hopefully for the life of the patient,” because these cells don’t divide
Doctors think they need to fix one tenth to one third of the cells to restore vision.