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Tests Suggest Scientists Achieved 1st “In Body” Gene Editing

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Tests Suggest Scientists Achieved 1st “In Body” Gene Editing

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The Associated Press – Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it is too soon to know if this will help.

Preliminary results suggest that two men with a rare disorder now have a corrective gene at very low levels, which may not be enough to make the therapy a success.

Still, it is a scientific milestone toward doctor’s hopes of editing DNA to treat diseases caused by faulty genes.

“This is a first step,” said Dr. Joseph Muenzer of the University of North Carolina at Chapel Hill, who helped test the treatment. “It’s just not potent enough.” Researchers are working on a stronger version of the treatment.

Gene editing is intended as a more precise way to do gene therapy, to disable a bad gene or supply a good one that is missing. The DNA changes do not pass to future generations, unlike the recent case of a Chinese scientist who claims to have edited twin girls’ genes when they were embryos.

The rare disorders being treated with gene editing is Hunter or Hurler syndrome, which is caused by a missing gene that makes an enzyme to break down certain sugar compounds. Without it, sugars build up and damage organs, often killing people in their teens.

In 2017, Brian Madeux of Arizona became the first person to try it. Through an IV, he received many copies of a corrective gene and an editing tool called zinc finger nucleases to insert it into his DNA.

Results on him and seven other Hunter patients, plus three with Hurler syndrome, suggest the treatment is safe, which was the main goal of these early experiments.

Blood tests detected slightly higher levels of the missing enzyme in a few of the Hunter patients but none of them reached normal levels. One patient had a larger increase but also showed signs that his immune system might be attacking the therapy.

More encouraging results were seen in Hurler patients—enzyme levels rose to normal in all three after treatment, tests on certain blood cells showed.

“This is very promising” for Hurler patients, said Dr. Paul Harmatz of UCSF Benioff Children’s Hospital Oakland, who presented those results.

None of the patients with either disease showed a sustained decline in urine levels of the troubling sugar compounds, though, and some other tests also did not detect intended effects of the therapy.

The key test will be stopping the patients’ weekly enzyme treatments to see if their bodies can now make enough of it on their own.

“It looks like it’s safe…that’s a very positive sign,” said one independent expert, Dr. Kiran Musunuru of the University of Pennsylvania. He called the early results promising but said “it’s hard to be sure it’s doing any good” until patients are studied longer.

“What they’re trying to do with gene editing is very challenging,” he said. “It’s much harder to make a correction or insert a gene” than to disable one.

Dr. Tyler Reimschisel of Vanderbilt University agreed.

“It’s not discouraging, it’s just early and on a small amount of people,” he said. “This is definitely a novel and innovative treatment” but it’s not clear if it’s going to help.

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