Early, partial results from a historic gene editing study indicate that the treatment may be safe and having at least some of its hoped-for effect.
However, it is too soon to know whether it will ultimately succeed.
The results, announced in September, are from the first human test of gene editing in the body – an attempt to permanently change someone’s DNA to cure a disease.
In this case, they are tackling a genetic disorder called Hunter syndrome that often kills people in their teens. In two patients who got a medium dose of the treatment, urine levels of large sugar compounds that are hallmarks of Hunter syndrome had fallen by half, on average, four months later – a possible sign the treatment is working.
Two others who got a low dose have seen little change in these sugars so far.
However, there’s no way to know yet whether the change are due to the gene editing.
