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Review

Stem cell therapy for muscular dystrophy

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Pages 1-9 | Published online: 03 Mar 2005

Bibliography

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  • ••This work demonstrates that Pax7 isrequired for the development of satellite cells, but not embryonic or fetal muscle cell lineages.
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  • ••This work shows that transplanted BMcells can participate in the regeneration of injured muscle in mice by first giving rise to the muscle satellite cell. The authors propose that BM cells can differentiate into non-haematopoietic tissues via a tissue-specific stem cell or precursor (such as the muscle satellite cell).
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  • •In this paper, the authors demonstrate that expression of a truncated utrophintransgene within the skeletal muscle of mice null for both dystrophin and utrophin rescues the clinical and pathological phenotype. This pharmacological approach alleviates the dystrophic pathology by compensating for the lack of dystrophin through utrophin.
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  • •Here, the authors demonstrate that intraperitoneal injections of blocking antibodies against myostatin in mix-mice lead to increased muscle mass and decreased muscle degeneration. The functional improvement of dystrophic muscle via myostatin blockade is one novel pharmacological approach to the treatment of DMD.
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  • •This paper describes the use of minimal functional dystrophin units which can be delivered via adeno-associated viral vectors. Delivery of minidystrophin via adeno-associated virus to mdxmice ameliorated the dystrophic pathology.
  • HARPER SQ, HAUSER MA, DELLORUSSO C et al.: Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat. Med. (2002) 8:253–261.
  • •This paper also demonstrates the promising potential of gene therapy using adeno-associated viruses to deliver micro-dystrophins to ameliorate the pathology present in mdx mice.
  • DE ANGELIS FG, STHANDIER O, BERARDUCCI B et al.: Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells. Proc. Natl. Acad. Sci. USA (2002) 99:9456–9461.
  • RANDO TA: Oligonucleotide-mediated gene therapy for muscular dystrophies. Neuromuscul Disord. (2002) 12\(Suppl. 1):555–560.
  • •This paper reviews the novel approach of using oligonucleotides to repair frameshift mutations in the dystrophin gene.

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