Nature Medicine
- 12, 175 - 177 (2006)
Published online: 29 January 2006; | doi:10.1038/nm1345
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathologyJulia Alter1, Fang Lou2, Adam Rabinowitz1, HaiFang Yin1, Jeffrey Rosenfeld3, Steve D Wilton4, Terence A Partridge1 & Qi Long Lu31
Muscle Cell Biology, MRC Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK. 2
School of Life Science, University of Hertfordshire, College Lane, Hatfield, Herts, AL10 9AB, UK. 3
Muscular Dystrophy Laboratory, Neuromuscular/ALS Center, Carolinas Medical Center, 1000 Blythe Boulevard, Charlotte, North Carolina 28231, USA. 4
Australian Neuromuscular Research Institute, Centre for Neuromuscular and Neurological Disorders, QEII Medical Centre University of Western Australia, Nedlands 6009, Western Australia.
Correspondence should be addressed to Qi Long Lu qi.lu@carolinashealthcare.org 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.
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