An opening in Duchenne muscular dystrophy: Persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice;Le saut d'exon thérapeutique: Un espoir pour les dystrophinopathies

Medecine/Sciences

Volumn 20, Issue 12, 2004, Pages 1163-1165

  Goyenvalle, Aurélie ^a   Vulin, Adeline ^a,b   Fougerousse, Françoise ^a   Leturcq, France ^c   Kaplan, Jean Claude ^c   Garcia, Luis ^a   Danos, Olivier ^a  

^a GÉNÉTHON   (France)

^b ECOLE NATIONALE VÉTÉRINAIRE D ALFORT   (France)

^c INSTITUT COCHIN   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MONOCLONAL ANTIBODY; PARVOVIRUS VECTOR; SPECTRIN;

DUCHENNE MUSCULAR DYSTROPHY; EXON; EXPRESSION VECTOR; GENE MUTATION; GENE THERAPY; NONHUMAN; NOTE; SARCOLEMMA; WESTERN BLOTTING;

PARVOVIRUS; VECTORS;

EID: 10944241811     PISSN: 07670974     EISSN: None     Source Type: Journal    
DOI: 10.1051/medsci/200420121163     Document Type: Note

References (9)

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2. Harper SQ, Hauser MA, DelloRusso C, et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med 2002; 8: 253-61.
3. Matsuo M, Masumura T, Nishio H, et al. Exon skipping during splicing of dystrophin mRNA precursor due to an intraexon deletion in the dystrophin gene of Duchenne muscular dystrophy kobe. J Clin Invest 1991; 87: 2127-31.
4. Aartsma-Rus A, Bremmer-Bout M, Janson AA, et al. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord 2002; 12 (suppl 1): S71-7.
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