“I’m old and having Hunter’s has done a lot of damage to my body,” said Madeux, right.
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Early, partial results from a historic gene-editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it's too soon to know whether it ultimately will succeed.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. A liver biopsy on one patient given a low dose of the therapy found no evidence that the gene editing had occurred, but Sangamo scientists said this dose is far below the level at which such signs had been detected in research on primates.Two other patients were given a middle dose that was twice what the first two patients received. NEXT STEPSTwo more patients have been given the highest dose being tested – 10 times the starting dose – for a total of six patients in the study.
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