U.S. 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 try to cure a disease.
Brian Madeux with Hunter syndrome gets IV infusion of gene and a genetic tool to splice it into his DNA.The experiment was done Monday in California on 44-year-old Brian. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.
Brian shared, “It’s kind of humbling” to be the first to test this.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.
“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.”That also means there’s no going back, no way to erase any mistakes the editing might cause.
Fewer than 10,000 people worldwide have these metabolic diseases, partly because many die very young. Those with Madeux’s condition, Hunter syndrome , lack a gene that makes an enzyme that breaks down certain carbohydrates. These build up in cells and cause havoc throughout the body.
Patients may have frequent colds and ear infections, distorted facial features, hearing loss, heart problems, breathing trouble, skin and eye problems, bone and joint flaws, bowel issues and brain and thinking problems.
Madeux, who now lives near Phoenix, is engaged to a nurse, Marcie Humphrey, who he met 15 years ago in a study that tested this enzyme therapy at UCSF Benioff Children’s Hospital Oakland, where the gene editing experiment took place.
He has had 26 operations for hernias, bunions, bones pinching his spinal column, and ear, eye and gall bladder problems.
Gene editing won’t fix damage he’s already suffered, but he hopes it will stop the need for weekly enzyme treatments.