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Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.
Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. Engelbeen S, et al. Nucleic Acid Ther. 2023 Dec;33(6):348-360. doi: 10.1089/nat.2023.0038. Nucleic Acid Ther. 2023. PMID: 38010230 Free PMC article.