Medical first: Doctors attempt to gene-edit a living patient’s DNA

A man may soon be forever free of the previously incurable disease he was born with 44 years ago. On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body’s cells.

From Madeux’s point of view, the revolutionary treatment appears anything but: It was a simple matter of getting hooked up to an IV, which delivered a gene editor into his bloodstream along with copies of a corrected gene to replace the mutation he carries that is responsible for the disorder.
“The infusion takes place for anywhere from two to three hours. And that’s it,” said Dr. Edward Conner, senior vice president and chief medical officer of Sangamo Therapeutics, maker of the new therapy. “We’re very hopeful.”
Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children's Hospital in Oakland, California.