Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to cure a disease. The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he 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’s working may come in a month; tests will show for sure in three months. If it’s successful, it 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 don’t 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 like cancer. READ MORE
