The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
Batra R, Nelles DA, Roth DM, Krach F, Nutter CA, Tadokoro T, Thomas JD, Sznajder LJ, Blue SM, Gutierrez HL, Liu P, Aigner S, Platoshyn O, Miyanohara A, Marsala M, Swanson MS, Yeo GW
Nat Biomed Eng. 2021 Feb;5(2):157-168. doi: 10.1038/s41551-020-00607-7. Epub 2020 Sep 14.
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