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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References
Arnett, A.L., Chamberlain, J.R. & Chamberlain, J.S. Curr. Opin. Genet. Dev. 19, 290–297 (2009).
van Deutekom, J.C. et al. N. Engl. J. Med. 357, 2677–2686 (2007).
Kinali, M. et al. Lancet Neurol. 8, 918–928 (2009).
Wheeler, T.M. et al. Science 325, 336–339 (2009).
Mann, C.J. et al. Proc. Natl. Acad. Sci. USA 98, 42–47 (2001).
Alter, J. et al. Nat. Med. 12, 175–177 (2006).
Yokota, T. et al. Ann. Neurol. 65, 667–676 (2009).
Miller, J.W. et al. EMBO J. 19, 4439–4448 (2000).
Liquori, C.L. et al. Science 293, 864–867 (2001).
Margolis, J.M., Schoser, B.G., Moseley, M.L., Day, J.W. & Ranum, L.P. Hum. Mol. Genet. 15, 1808–1815 (2006).
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).
Svasti, S., et al. RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. Proc. Natl. Acad. Sci. USA. 164, 1205–1210 (2009).
Goyenvalle, A., Babbs, A., van Ommen, G.-J., Garcia, L. & Davies, K.E. Mol. Ther. 17, 1234–1240 (2009).
Gregorevic, P. et al. Nat. Med. 10, 828–834 (2004).
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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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DOI: https://doi.org/10.1038/nm0210-170
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