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MBL Senior Scientist Joshua Rosenthal, an expert in RNA editing, is quoted in this article.

Two RNA-editing therapies for genetic diseases have in the past few months gained approval for clinical trials, raising hopes for safer treatments.

RNA editing is gaining momentum. After decades of into how to manipulate this complex molecule, at least three therapies based on RNA editing have either entered clinical trials or received approval to do so. They are the first to reach this milestone.

Proponents of RNA editing have long argued that it could be a safer and more flexible alternative to , but it poses substantial technical problems.

The launch of human trials signals the growing maturity and acceptance of the field, scientists say. “There’s a much greater understanding of RNA technology, and that’s been partially enhanced by the RNA vaccine and the COVID pandemic,” says Andrew Lever, a biologist at the University of Cambridge, UK. “RNA is now seen as a very important therapeutic molecule.”

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