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Why gene editing isn’t ready for treating human disease: It’s not ‘efficient enough’

| | February 28, 2019

Gene editing will likely always come with a bit of risk; when you’re cutting and pasting DNA in millions of cells, extremely rare events can’t be avoided.

In the new work, spread across three papers, researchers tackle two genetic diseases that seem to tick most of the boxes for “worth the risk.” The disease are Duchenne muscular dystrophy (DMD) and Hutchinson–Gilford progeria syndrome (HGPS).

If these are the sorts of disorders we could treat with gene editing, how close are we to doing so? To find out, researchers decided to test out the systems in mice.

Overall, the three papers present a consistent picture. The technology works, it’s possible to fix genetic defects through gene editing, and the fixes can have an effect on health. But the editing isn’t currently efficient enough to provide anything more than some partial help for people with these diseases. And that help comes with some significant risks: errors in editing, activity in the wrong cells, and an immune response that limits future interventions. We’ve got a lot of work ahead of us before this is ready for the clinic.

Related article:  First gene-editing experiments on adults suffering from rare metabolic disorders offer promising early results

Read full, original post: Gene editing still has a few bugs in the system

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