Abstract
Antisense oligoribonucleotides (AONs) are able to induce exon skipping in the dystrophin hnRNA; a successful first-in-man trial has recently been completed on DMD and a multicentric trial is approaching .The objectives of our project are (i) pre-screening characterization of myogenic cells from DMD boys with dystrophin deletions eligible for either exon 44 or 51 skipping approach to test their suitability to the trial; (ii) identification of genomic and transcriptomic biomarkers to be possibly used to monitor the treatment efficacy and safety.
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