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Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy
This study assesses exon 53 skipping in Duchenne muscular dystrophy (DMD) using locked nucleic acid (LNA)-modified antisense oligonucleotides (AONs) in a humanized DMD mouse model. While certain LNA-AONs (LNA-FRNA, LNA-2′OMe) achieved high exon skipping, actual dystrophin restoration was minimal due to RNA interference issues affecting cDNA synthesis. Optimizing AON designs, RNA analysis methods, and alternative exon targets is crucial for improving therapeutic efficacy in DMD exon skipping therapies.
