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03/05/2019

GENE EDITING IS TRICKIER THAN EXPECTED—BUT FIXES ARE IN SIGHT

External Link - Source: Wired

OF ALL THE big, world-remaking bets on the genome-editing tool known as Crispr, perhaps none is more tantalizing than its potential to edit some of humanity’s worst diseases right out of the history books. Just this week, Crispr Therapeutics announcedit had begun treating patients with an inherited blood disorder called beta thalassemia, in the Western drug industry’s first test of the technology for genetic disease. But despite the progress, there remain a host of unknowns standing in the way of Crispr-based medicines going mainstream, chief among them safety.

That’s because the classic, most widely used version of Crisprworks by slicing open a strand of DNA in a specific spot in the genome and letting the cell stitch it back together. The major concern is that an army of DNA-breaking enzymes might sometimes wander astray and cause unintended mutations in places it shouldn’t.

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