Jan 31, 2006

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature Medicine
Julia AlterQi Long Lu

Abstract

For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse, with resulting improvement in muscle function. Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD.

  • References15
  • Citations265

References

Mentioned in this Paper

Brain Diseases
DMD gene
Exons
Intravenous Injections
Soleus Muscle Structure
Muscle Function
Oligodeoxyribonucleotides, Antisense
Dystrophin
Oligonucleotides
Genetic Therapy, Somatic

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