Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice

This publication has 39 references indexed in Scilit:

• Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
  Molecular Therapy, 2010
• Dosing Regimen Has a Significant Impact on the Efficiency of Morpholino Oligomer-Induced Exon Skipping in mdx Mice
  Human Gene Therapy, 2009
• Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
  Nature Medicine, 2006
• Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
  Proceedings of the National Academy of Sciences, 2004
• Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
  Human Molecular Genetics, 2003
• Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
  Nature Medicine, 2003
• Somatic reversion/suppression of the mouse mdx phenotype in vivo
  Journal of the Neurological Sciences, 1990
• Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
  Nature, 1990
• An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
  Genomics, 1988
• X chromosome-linked muscular dystrophy (mdx) in the mouse.
  Proceedings of the National Academy of Sciences, 1984