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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping.
Mol Ther Methods Clin Dev. 2021 Mar 23;21:325-340. doi: 10.1016/j.omtm.2021.03.014. eCollection 2021 Jun 11.
Mol Ther Methods Clin Dev. 2021.
PMID: 33898631
Free PMC article.
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