CRISPR update calms fears about off-target editing effects

crispr

The fear that CRISPR-based genome repair for preventing or treating genetic diseases will be derailed by โ€œediting gone wildโ€ has begun to abate, scientists who are developing the technique say.

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Aย studyย last year involving just three mice raised fears that CRISPR-Cas9, the classic version of the genome editor, might be a big, stupid bloodhound. Scientists at Stanford University reported they found hundreds of off-target effects, which they said was โ€œof concernโ€: Messing with a tumor-suppressor gene could cause cancer, while other edits โ€œcould be detrimental to key cellular processes,โ€ they wrote.

The stock prices of several CRISPR companies swooned, butย CRISPR experts immediately pounced. In addition to using so few mice, the study, they said, didnโ€™t account for the fact that DNA differences between the two CRISPRโ€™d mice and the control mouse might simfply have reflected different ancestry.

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Scientists led by Dr. Keith Joung of Massachusetts General Hospital, however, have discovered a way to drastically minimize that bystander effect. According to an unpublishedย paper…they tweaked the base editor to, in essence, change that C only if the letter before it is, say, a T. Itโ€™s akin to priming the bloodhound to find suspects wearing Nikes only if the aroma of Dockers is also around.

Read full, original post:ย CRISPR โ€˜gone wildโ€™ has made stocks swoon, but studies show how to limit off-target editing

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