Early Results Give Hope For Human Gene Editing to Change DNA

(PHOENIX) — 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. The results announced Wednesday 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, 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. There’s no way to know yet whether the change in the middle-dose patients is due to the gene editing or something else, but the fact their sugars have declined consistently since treatment suggests it might be. “I cannot absolutely say it’s a treatment effect” but the drop is “really encouraging,” said the study leader, Dr. Joseph Muenzer of the University of North Carolina, Chapel Hill. The main goal of early treatment studies is to test safety, though researchers also look for hints that the therapy is working. Muenzer gave the results at a conference in Greece and consults for the treatment’s maker, California-based Sangam...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Disease onetime Source Type: news