A super-sharp gene editing tool could tackle high cholesterol and other common ailments

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A super-sharp gene editing tool could tackle high cholesterol and other common ailments
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Helping Americans avoid heart disease, one DNA base pair at a time.

. But for all the good it’s done for the world, CRISPR/Cas9 is not perfect. While the technology allows experts to make detailed edits in a living organism, it has not been precise enough to make the exact edits needed to reverse or prevent genetic diseases. There’s also concern that multiple or incorrect edits could harm a person’s cells, introducing mutations in the selected DNA sequence that raise, say, the risk of cancer.

Cue in CRISPR’s cousin. As seen in the cholesterol trial, the new method cuts only one DNA strand instead of two, and uses another enzyme attached to Cas9 that works like a pencil to make a small edit to the genetic code. The base-editing enzyme chemically rewrites one letter of DNA to change it to another.

“The number one cause of mortality in the world is heart attack,” said Sekar Kathiresan, CEO of Verve Therapeutics, the biotechnology company that created the base-editing technique, per. “If you are going to give a medicine that makes you avoid a heart attack, people are going to live longer.”

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