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New gene-editing treatment might help treat a rare disorder, hints first human test

External Link - Source: Science

The first test of a new gene-editing tool in people has yielded early clues that the strategy—an infusion that turns the liver into an enzyme factory—could help treat a rare, inherited metabolic disorder. Today, the biotech company Sangamo Therapeutics in Richmond, California, reported data suggesting that two patients with Hunter syndrome are now making small amounts of a crucial enzyme that their bodies previously could not produce. But the company is still a long way from providing evidence that the new method can improve Hunter patients’ health.

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