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Scientists working toward reversible kind of gene editing

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Scientists are altering a powerful gene-editing technology in hopes of one day fighting diseases without making permanent changes to people’s DNA. The trick: Edit RNA instead, the messenger that carries a gene’s instructions.

“If you edit RNA, you can have a reversible therapy,” important in case of side effects, said Feng Zhang of the Broad Institute of MIT and Harvard, a gene-editing pioneer whose team reported the new twist Wednesday in the journal Science.

It’s a biological cut-and-paste tool that lets researchers spot a gene defect inside living cells and use molecular “Scissors” to snip that spot, either deleting, repairing or replacing the affected gene.

DNA repair is harder to achieve in certain cells, such as brain and muscle cells, than in others, such as blood cells-so targeting RNA may offer an important alternative, said University of California, San Diego, professor Gene Yeo, who wasn’t involved in Wednesday’s study.

Editing RNA’s instructions should result in temporary fixes to abnormal protein production, Zhang explained.

Zhang’s team examined relatives in the Cas protein family and found one, Cas13, that could target RNA instead. The researchers engineered a Cas13 variety so it sticks to RNA instead of cutting it.

Explore further: New CRISPR tool targets RNA in mammalian cells.


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