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cystic fibrosis

nonsense mutations

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Scientists Stopped Trying to Fix the Gene. They Fixed the Reader

Sep 1, 2026

•

5 min read

Scientists Stopped Trying to Fix the Gene. They Fixed the Reader

Some genetic diseases are caused by a single misplaced full stop in the middle of an instruction. The usual answer is to correct the error, which means editing DNA. A team at the University of Toronto did something else: they left the typo in place and rebuilt the molecule that reads it, then delivered the fix as something you breathe in. Here is how readthrough works, and what it would take for it to reach patients.

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