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If the genome-editing system CRISPR-Cas9 is biology’s precise, disciplined, Swiss army knife, its march toward the clinic is more like the roller coaster from hell. One moment it’s riding high with the promise of curing devastating genetic diseases, then its prospects plummet after the discovery of previously unsuspected risks, and the next moment it turns out those risks are either overblown or avoidable. Buy another ticket and get back on board.

On Thursday, scientists led by one of the world’s foremost gene therapy experts reported a way around one of the more worrisome obstacles: that CRISPR’d cells might be prone to becoming cancerous, as two 2018 studies suggested.

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  • Seems like a reasonable solution for ex-vivo, but AAV-delivered in vivo CRISPR still problematic?

  • So they found a patch for the CRISPR problem? That should do it! Nothing to worry about now!

  • How long before this mouse fix for CRISPR is incorporated into human trials? Sangamo’s ZFN’s already edit without p53 mutation. They are in the clinic for beta-thalassemia and sickle cell both partnered with Sanofi.

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