The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
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Nat Biomed Eng. 2021 Feb;5(2):157-168. doi: 10.1038/s41551-020-00607-7. Epub 2020 Sep 14.
Nat Biomed Eng. 2021.
PMID: 32929188
Free PMC article.