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DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
Developing tissue specific antisense oligonucleotide-delivery to refine treatment for Duchenne muscular dystrophy
BMP signaling in vascular and heterotopic bone diseases
Refinement of antisense oligonucleotide mediated exon skipping as therapy for Duchenne muscular dystrophy