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Cell Press, Molecular Therapy - Methods and Clinical Development, (21), p. 325-340, 2021

DOI: 10.1016/j.omtm.2021.03.014

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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Journal article published in 2021 by Tabatha R. Simmons, Tatyana A. Vetter ORCID, Nianyuan Huang, Adeline Vulin-Chaffiol, Nicolas Wein ORCID, Kevin M. Flanigan ORCID
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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