The most common gene editing technique, CRISPR-Cas9, only modifies DNA. That’s helpful in most cases, but it means that you can’t use it to tackle RNA-based diseases. After plenty of talk about editing RNA, researchers have developed a new RNA-oriented technique (RCas9) that can correct the molecular errors which lead to diseases like hereditary ALS and Huntington’s.
The team achieved its feat using guide RNA that steers the editing enzyme toward a matching sequence of target RNA molecules. In the lab, it’s highly effective — it fixed nearly all of the broken RNA targets in muscle cells, leaving cells that were far healthier.
…[T]his could prove to be hugely important for treating these RNA diseases. Right now, there aren’t any therapies that would stall the onset of diseases like ALS. This wouldn’t likely represent a cure, but it could give patients full functionality for longer before the effects of their diseases set in.
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