February 8 2019

Doctoring DNA: Scientists Achieved FIRST ‘In-Body’ Gene Editing

Playing ‘Doctor God’: Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to…

Playing ‘Doctor God’: Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, although it’s 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’s a scientific milestone toward one day doctoring DNA to treat many 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.”

He gave the results Thursday at a conference in Orlando, Florida, and has consulted for the therapy’s maker, California-based Sangamo Therapeutics. 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’s missing. Trying it in adults to treat diseases is not controversial and 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.

“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.

Sangamo’s president, Dr. Sandy Macrae, said a more potent version is being manufactured. (Read More)