From The Guardian:
Scientists have tried editing a gene inside the body for the first time, in a bold attempt to tackle an incurable a disease by permanently changing a patient’s DNA.
On Monday in California, 44-year-old Brian Madeux intravenously received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.
“It’s kind of humbling to be the first to test this,” said Madeux, who has a metabolic disease called Hunter syndrome. “I’m willing to take that risk. Hopefully it will help me and other people.”
Signs of whether it is working may come in a month; tests will confirm in three months.
If successful, the new technique could give a major boost to the fledgling field of gene therapy. Scientists have edited people’s genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that do not involve editing DNA.
But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can’t control where it inserts in the DNA, possibly causing a new problem, such as cancer.
This time, the genetic tinkering is happening in a precise way inside the body – like sending a miniature surgeon along to place the new gene in exactly the right location.