Gene therapy has been around for a while, but U.S. scientists are taking it to a new level with gene editing inside the human body. Previously, therapies were administered after alteration in the lab and offered treatment of symptoms for limited conditions. If successful, this breakthrough holds the possibility of curing disease by permanently altering DNA within the body.
“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”
The first patient to participate in the experiment is 44-year-old Brian Madeux, who underwent treatment in California on Monday.
“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.”
Scientists said signs the therapy is working could be seen in as little as a month, and testing at three months will offer conclusive results. Initial tests will involve 30 adult participants, but the end goal is to treat children before disease has caused much damage.
“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.