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The first attempt at using genome editing to treat and cure patients with a rare, inherited disease has produced disappointing results in a small clinical trial.

Despite the setback, Sangamo Therapeutics (SGMO), the biotech developing the genome-editing therapy, intends to keep trying. Transient improvements observed in a single patient suggest a more potent version of its treatment — already being readied for use — might be more effective, the company said.


For now, however, reality is sobering: A landmark attempt at using groundbreaking technology to repair disease-causing DNA has fallen short of high expectations.

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  • Hi Adam, I’m a great fan of yours!
    Thanks for the summary, but I wonder what is your opinion? Based on the observed data, do you think it could be solved by dosing 1e14 / using ZFN2? Or more luck in children vs. adults? Simply to early to judge anything? Thanks for giving your take!


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