The Journal of Gene Medicine

نویسندگان

  • Hans A. Heemskerk
  • Christa L. de Winter
  • Sjef J. de Kimpe
  • Petra van Kuik-Romeijn
  • Niki Heuvelmans
  • Gerard J. Platenburg
  • Gert-Jan B. van Ommen
  • Judith C. T. van Deutekom
  • Annemieke Aartsma-Rus
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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تاریخ انتشار 2009