Thursday, October 26, 2017

New CRISPR tool alters RNA for wider gene editing applications



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US researchers announced Wednesday that they have expanded the powerful gene-editing CRISPR tool to also edit RNA. To prove its therapeutic potential, the scientists in the lab isolated a cell from a patient suffering from hereditary haemochromatosis (HHC), a crippling disease caused by the excessive iron buildup and treated by bleeding patients.

Nothing's quite ready for use in actual patients, but the reports show there's more than one way to fix a broken genetic code. Not only did the procedure have a much higher success rate than other gene-editing techniques, there were virtually no side-effects, such as unwanted DNA duplications or deletions. "This new ability to edit RNA opens up more potential opportunities to recover that function and treat many diseases, in nearly any kind of cell".

The greater precision of the technique should enable finer genetic manipulations than ever before, introducing fewer random errors carried over from adjacent nucleobases that are inevitably copied over with the targeted DNA. For this first study, the team manipulated the genetic code so that the defective gene was changed.

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