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.
About:
The studies involve men with Hunter or Hurler Syndrome, diseases 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.
Working:
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.
Joseph Muenzer of the University of North Carolina at Chapel Hill, helped test the treatment, with the help of the therapy's maker, California-based Sangamo Therapeutics.
Findings:
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.
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, a first step toward one day doctoring DNA to treat many diseases caused by faulty genes.
Significance:
In body 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.
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