Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Alter, Julia, Lou, Fang, Rabinowitz, Adam, Yin, HaiFang, Rosenfeld, Jeffrey, Wilton, Steve D, Partridge, Terence A and Lu, Qi Long
(2006)
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.
pp. 175-7.
ISSN 1078-8956
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.
Item Type | Article |
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Uncontrolled Keywords | Animals; Base Sequence; Drug Administration Schedule; Dystrophin; Gene Expression Regulation; Gene Therapy; Humans; Injections, Intravenous; Male; Mice; Mice, Inbred C57BL; Mice, Inbred mdx; Muscle, Skeletal; Muscular Dystrophy, Animal; Muscular Dystrophy, Duchenne; Oligodeoxyribonucleotides, Antisense |
Date Deposited | 14 Nov 2024 10:27 |
Last Modified | 14 Nov 2024 10:27 |