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Review
. 2004 Sep;5(9):872-6.
doi: 10.1038/sj.embor.7400221.

Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment

Affiliations
Review

Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment

Kristen J Nowak et al. EMBO Rep. 2004 Sep.

Abstract

Third in Molecular Medicine Review Series

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Figures

Figure 1
Figure 1
Summary of the wide range of approaches being used to treat Duchenne muscular dystrophy. IGF1, insulin-like growth factor 1; NOS, nitric oxide synthase; TNF-α, tumour necrosis factor-α.
Figure 2
Figure 2
The dystrophin-associated protein complex in muscle linking the internal cytoskeleton to the extracellular matrix. NOS, nitric oxide synthase.
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References

    1. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, Van Ommen GJ, den Dunnen JT, van Deutekom JC (2003) Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet 12: 907–914 - PubMed
    1. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, van Ommen G, den Dunnen JT, van Deutekom JCT (2004) Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet 74: 83–92 - PMC - PubMed
    1. Arakawa M et al. (2003) Negamycin restores dystrophin expression in skeletal muscle of mdx mice. J Biochem 134: 751–758 - PubMed
    1. Bachrach E, Li S, Perez AL, Schienda J, Liadaki K, Volinski J, Flint A, Chamberlain J, Kunkel LM (2004) Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells. Proc Natl Acad Sci USA 101: 3581–3586 - PMC - PubMed
    1. Barresi R et al. (2004) LARGE can functionally bypass α-dystroglycan glycosylation defects in distinct congenital muscular dystrophies. Nat Med 10: 696–703 - PubMed

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