Monday, November 13 found one man the first ever to receive an edited gene. 44-year-old Brian Madeux received the treatment in a California hospital, "intravenously [receiving] billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot."
Scientists hope that by editing genes inside the human body, they can begin to cure some of the incurable diseases. This will, of course, mean permanently altering a patient's DNA.
“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.”
According to The Guardian, the treatment may start working in as little as a month, and tests will confirm within three months. If the treatment is successful "the new technique could give a major boost to the fledgling field of gene therapy."
Currently, editing DNA directly can only be used for a few types of diseases. Some of the edits may not last, while others may cause new problems, since they "can’t control where it inserts in the 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 the therapy for two metabolic diseases and haemophilia. “It becomes part of your DNA and is there for the rest of your life.”
Dr. Eric Topol of the Institute of San Diego says the promise of this new method is too great to ignore, even if there are certain risks associated with it. “So far there’s been no evidence that this is going to be dangerous,” he said. “Now is not the time to get scared.”
In order to make sure that the gene-editing tool, Crispr-Cas9, is safe, they will need to test upwards of 30 adults. But Madeux's treatment involved a new tool called "zinc finger nucleases." The tool works "like molecular scissors that seek and cut a specific piece of DNA."
Gene editing cannot reverse damage that has already been sustained, but it can prevent further damage and the need for hospital visits.
Although there are risks with his specific procedure, Madeaux is optimistic.