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Nature Research, Nature Biomedical Engineering, 2(5), p. 157-168, 2020

DOI: 10.1038/s41551-020-00607-7

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The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

Journal article published in 2020 by Ranjan Batra, David A. Nelles, Daniela M. Roth, Florian Krach, Curtis A. Nutter, Takahiro Tadokoro ORCID, James D. Thomas ORCID, Łukasz J. Sznajder ORCID, Steven M. Blue ORCID, Haydee L. Gutierrez, Patrick Liu, Stefan Aigner, Oleksandr Platoshyn ORCID, Atsushi Miyanohara, Martin Marsala ORCID and other authors.
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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