This article appeared in Discover’s annual state of science issue as “CRISPR Gets Under the Skin.” Support our science journalism by becoming a subscriber.
For the first time, scientists have injected the CRISPR-Cas9 gene-editing tool into a human patient as part of a clinical trial. It marks another milestone for human gene editing, and a step toward bringing gene therapies to wider patient populations.
The pharmaceutical companies Allergan and Editas Medicine partnered with Oregon Health & Science University for the trial, named BRILLIANCE, which aims to treat a form of congenital eye disease known as LCA10. In the trial, scientists are injecting instructions for the CRISPR gene editor, encapsulated in the shell of a deactivated virus, into patients’ eyes. There, if all goes well, the tool will cut out a problematic gene and restore their vision.
Though CRISPR has been used in the past to treat patients, those trials involved ...
