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A new genome editing method may overcome critical barriers to correcting disease-causing genetic mutations. The approach draws on the popular clustered regularly interspaced short palindromic repeat (CRISPR)–associated 9 (Cas9) technology but avoids some of its undesired effects on DNA. In principle, the technique—called prime editing—could correct an estimated 89% of genetic variants known to be associated with human diseases.
The strategy, described recently in a study published in Nature, relies on prime editors—an altered form of the Cas9 protein and an RNA that together orchestrate a series of DNA targeting, writing, and repair steps that result in an edit. Unlike with typical CRISPR-Cas9 technology, the prime editor Cas9 doesn’t make double-stranded cuts in DNA, which can lead to uncontrolled DNA insertions and deletions at the cut site. Instead, the altered Cas9 only snips a single strand of the double helix.
Hampton T. DNA Prime Editing: A New CRISPR-Based Method to Correct Most Disease-Causing Mutations. JAMA. 2020;323(5):405–406. doi:10.1001/jama.2019.21827
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