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Early results boost hopes for gene editing

Posted at 6:36 AM, Sep 05, 2018
and last updated 2018-09-05 09:41:17-04

Early 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 tests of gene editing in the body, an attempt to permanently change someone's DNA to cure a disease. Doctors treated four people with a genetic disorder called Hunter syndrome.
Two patients had a drop in troubling sugar compounds, a possible sign that the treatment helped. Two other patients who were given a much lower dose have not seemed to benefit so far.
Results were given at a conference in Greece and announced by the treatment's maker, California-based Sangamo (SANG-uh-mow) Therapeutics.