Free Access
Issue
Med Sci (Paris)
Volume 24, Number 2, Février 2008
Page(s) 215 - 218
Section Dernière heure
DOI https://doi.org/10.1051/medsci/2008242215
Published online 15 February 2008
  1. Van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007; 357 : 2677–86. [Google Scholar]
  2. Kaplan JC, Delpech M. Biologie moléculaire et médecine, 3e ed. Paris : Flammarion Médecine-Sciences, 2007 : 401–16. [Google Scholar]
  3. Van Deutekom JC, Van Ommen G. Advances in Duchenne muscular dystrophy gene therapy. Nat Med 2003; 4 : 774–83. [Google Scholar]
  4. Goyenvalle A, Vulin A, Fougerousse F, et al. Le saut d’exon thérapeutique : un espoir pour les dystrophinopathies. Med Sci (Paris) 2004; 20 : 1163–5. [Google Scholar]
  5. Benchaouir R, Meregalli M, D’Antonio G, et al. Réhabilitation de la dystrophine humaine dans le modèle scid/mdx après greffe de cellules souches de patients DMD corrigées par saut d’exon. Med Sci (Paris) 2008; 24 : 99–101. [Google Scholar]
  6. Monaco AP, Bertelson CJ, Liechti-Gallati S, et al. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988; 2 : 90–5. [Google Scholar]
  7. England S, Nicholson L, Johnson M, et al. Very mild muscular dystrophy associated with the deletion of 46 % of dystrophin. Nature 1990; 343 : 180–2. [Google Scholar]
  8. 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. [Google Scholar]
  9. Van Deutekom JC, Bremmer-Bout M, Janson AA, et al. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet 2001; 10 : 1547–54. [Google Scholar]
  10. Aartsma-Rus A, Janson AA, Kaman WE, et al. Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet 2004; 74 : 83–92. [Google Scholar]
  11. Aartsma-Rus A, Kaman WE, Weij R, et al. Exploring the frontiers of therapeuticexon skipping for Duchenne muscular dystrophy by double targetingwithin one or multiple exons. Mol Ther 2006; 14 : 401–7. [Google Scholar]
  12. Goyenvalle A, Vulin A, Fougerousse F, et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 2004; 306 : 1796–9. [Google Scholar]
  13. Lu QL, Rabinowitz A, Chen YC, et al. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 2005; 102 : 198–203. [Google Scholar]
  14. McClorey G, Moulton HM, Iversen PL, et al. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther 2006; 13 : 1373–81. [Google Scholar]
  15. Alter J, Lou F, Rabinowitz A, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006; 12 : 175–7. [Google Scholar]
  16. Benchaouir R, Meregalli M, Farini A, et al. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stemcells into dystrophic mice. Cell Stem Cell 2007; 1 : 644–57. [Google Scholar]
  17. Béroud C, Tuffery-Giraud S, Matsuo M, et al. Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy. Hum Mutat 2007; 28 : 196–202. [Google Scholar]
  18. Welch EM, Barton ER, Zhuo J, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007; 447 : 87–91. [Google Scholar]

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