Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD.

9 , 20 , 22 , 23 Building on these promising animal studies, ex-miRNAs were measured in the serum of DMD patients treated with eteplirsen (a naked phosphorodiamidate morpholino oligonucleotide [PMO] antisense oligonucleotide designed to induce skipping of DMD exon 51) for 12 weeks, whereby a trend toward therapeutic restoration was observed that did not reach statistical significance. 24 Importantly, achieving efficient restoration of dystrophin protein and accurate measurement of its expression in human dystrophic muscle remain significant challenges for the field. 25 The biological and clinical significance of altered extracellular sRNA levels are currently not well understood.

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