Neuromuscular Disorders
Volume 18, Issue 11 , Pages 904-905 , November 2008

Dystrophin knockdown mice suggest that early, transient dystrophin expression might be enough to prevent later pathology

Received 5 June 2008 ,Revised 5 June 2008 ,Accepted 24 June 2008.

References 

  1. Ghahramani Seno MM, Graham IR, Athanasopoulos T, et al. RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology. Hum Mol Genet. 2008;May 28: E-pub ahead of print
  2. Ahmad A, Brinson M, Hodges BL, et al. Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for Duchenne muscular dystrophy. Hum Mol Genet. 2000;9:2507–2515
  3. van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med. 2007;357:2677–2686
  4. Alter J, Lou F, Rabinowitz A, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med. 2006;12:175–177
  5. Li D, Yue Y, Duan D. Preservation of muscle force in mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein. Am J Pathol. 2008;172:1332–1341
  6. Knowlton KU. CVB infection and mechanisms of viral cardiomyopathy. Curr Top Microbiol Immunol. 2008;323:315–335

PII: S0960-8966(08)00537-3

doi: 10.1016/j.nmd.2008.06.371

Neuromuscular Disorders
Volume 18, Issue 11 , Pages 904-905 , November 2008

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