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Muscling in: Gene therapies for muscular dystrophy target RNA

  • Between Bedside and Bench
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Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to Bench', Simone Hettmer and Amy J. Wagers examine the implications of clinical studies describing a type of rhabdomyosarcoma that resembles acute leukemia. The findings dovetail with other studies suggesting that some of these cancers might originate outside of muscle tissue and highlight the need for a better understanding of the cells that give rise to this condition.

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Figure 1: Exon-skipping strategies using antisense oligonucleotides.

References

  1. Arnett, A.L., Chamberlain, J.R. & Chamberlain, J.S. Curr. Opin. Genet. Dev. 19, 290–297 (2009).

    Article  CAS  Google Scholar 

  2. van Deutekom, J.C. et al. N. Engl. J. Med. 357, 2677–2686 (2007).

    Article  CAS  Google Scholar 

  3. Kinali, M. et al. Lancet Neurol. 8, 918–928 (2009).

    Article  CAS  Google Scholar 

  4. Wheeler, T.M. et al. Science 325, 336–339 (2009).

    Article  CAS  Google Scholar 

  5. Mann, C.J. et al. Proc. Natl. Acad. Sci. USA 98, 42–47 (2001).

    Article  CAS  Google Scholar 

  6. Alter, J. et al. Nat. Med. 12, 175–177 (2006).

    Article  CAS  Google Scholar 

  7. Yokota, T. et al. Ann. Neurol. 65, 667–676 (2009).

    Article  Google Scholar 

  8. Miller, J.W. et al. EMBO J. 19, 4439–4448 (2000).

    Article  CAS  Google Scholar 

  9. Liquori, C.L. et al. Science 293, 864–867 (2001).

    Article  CAS  Google Scholar 

  10. Margolis, J.M., Schoser, B.G., Moseley, M.L., Day, J.W. & Ranum, L.P. Hum. Mol. Genet. 15, 1808–1815 (2006).

    Article  CAS  Google Scholar 

  11. Li, L.B., Yu, Z., Teng, X. & Bonini, N.M. RNA toxicity is a component of ataxin-3 degeneration in Drosophila. Nature 453, 1107–1111 (2008).

    Article  CAS  Google Scholar 

  12. Svasti, S., et al. RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. Proc. Natl. Acad. Sci. USA. 164, 1205–1210 (2009).

    Article  Google Scholar 

  13. Goyenvalle, A., Babbs, A., van Ommen, G.-J., Garcia, L. & Davies, K.E. Mol. Ther. 17, 1234–1240 (2009).

    Article  CAS  Google Scholar 

  14. Gregorevic, P. et al. Nat. Med. 10, 828–834 (2004).

    Article  CAS  Google Scholar 

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Correspondence to Joel R Chamberlain or Jeffrey S Chamberlain.

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Chamberlain, J., Chamberlain, J. Muscling in: Gene therapies for muscular dystrophy target RNA. Nat Med 16, 170–171 (2010). https://doi.org/10.1038/nm0210-170

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