In a world-first trial, scientists in California edited DNA inside the body in at attempt to cure a genetic disease.
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The patient is 44-year-old Brian Madeux, who has a metabolic disease called Hunter syndrome. During a three-hour IV infusion on Monday, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in an exact location, the Associated Press reported.
Indications of whether the procedure is working may become apparent in a month, and tests will provide a definitive answer in three months.
Until now, scientists have edited people's DNA by altering cells in the lab and then returning them to patients. There are also other types of gene therapies that don't involve editing DNA, the AP reported.
If successful, this new approach could be a major advance for the emerging field of gene therapy.
"We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending," Dr. Sandy Macrae, president of California-based Sangamo Therapeutics, told the AP. "It becomes part of your DNA and is there for the rest of your life."
The company is testing this method for two metabolic diseases and hemophilia.
There is no way to reverse mistakes that might occur while editing DNA within the body.
"You're really toying with Mother Nature" and there are unknown risks, independent expert Dr. Eric Topol of the Scripps Translational Science Institute in San Diego, told the AP. But these studies should continue because they target incurable diseases, he added.
Animal tests were very promising and protections are in place to help ensure safety, according to Dr. Howard Kaufman, a scientist on the National Institutes of Health panel that approved the studies.
The promise of this type of gene editing is too great to ignore, he told the AP.
"So far there's been no evidence that this is going to be dangerous," Kaufman said. "Now is not the time to get scared."
People with Hunter syndrome lack a gene that makes an enzyme that breaks down certain carbohydrates, leading them to accumulate in cells and cause a range of issues, including frequent colds and ear infections, hearing loss, heart and breathing trouble, skin and eye problems, bone and joint afflictions, bowel concerns, and brain and thinking problems, the AP reported.
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