The Journal of Gene Medicine
نویسندگان
چکیده
Background Antisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne into a milder Becker muscular dystrophy phenotype. In vivo studies are mainly performed using 2′-O-methyl phosphorothioate (2OMePS) or morpholino (PMO) AONs. These compounds were never directly compared.
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