CRISPR gene-editing tool used inside humans for the first time
The first trial of a CRISPR-based therapy to treat inherited blindness.
Jackson RyanFormer Science Editor
Jackson Ryan was CNET's science editor, and a multiple award-winning one at that. Earlier, he'd been a scientist, but he realized he wasn't very happy sitting at a lab bench all day. Science writing, he realized, was the best job in the world -- it let him tell stories about space, the planet, climate change and the people working at the frontiers of human knowledge. He also owns a lot of ugly Christmas sweaters.
"This dosing is a truly historic event -- for science, for medicine, and most importantly for people living with this eye disease," said Cynthia Collins, president and CEO of Editas Medicine, a gene-editing company headquartered in Massachusetts.
The breakthrough trial aims to test an experimental treatment for the genetically-inherited disease Leber congenital amaurosis 10. The disease is caused by a faulty gene that causes blindness from birth or during the first few months of life and affects around one in every 40,000 births. There are currently no approved treatment options.
The first patient in the trial received a dose of the experimental drug, called AGN-151587, via an injection in the eye. The idea is that it delivers the gene-editing tool CRISPR directly to cells in the eye which are affected by the genetic disease. CRISPR is able to find its way into those cells and correct the gene -- a cut-and-paste scenario that sees a tiny DNA edit made to remove the mutation.
Importantly, the CRISPR edit is permanent, which means patients may only need a single dose and be set for life.
The trial is expected to enroll 18 patients in total and will look at different doses of the experimental drug, refining how much is necessary to achieve the goal of reversing blindness -- without any side effects. Information about the first patient is scant, with researchers staying silent on patient information and when the surgery officially occurred.
Watch this: CRISPR explained with crisps (and assorted snacks)