A new study shows that normalizing the number of CUG repeat–containing DMPK transcripts in a mouse model of myotonic dystrophy reverses the myotonia and cardiac conduction defects. This discovery suggests new therapeutic approaches for the disease.
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References
Gatchel, J.R. & Zoghbi, H.Y. Nat. Rev. Genet. 6, 743–755 (2005).
Wang, J. et al. Hum. Mol. Genet. 4, 599–606 (1995).
Timchenko, L.T. Am. J. Hum. Genet. 64, 360–364 (1999).
Mankodi, A. et al. Science 289, 1769–1772 (2000).
Seznec, H. et al. Hum. Mol. Genet. 10, 2717–2726 (2001).
Taneja, K.L., McCurrach, M., Schalling, M., Housman, D. & Singer, R.H. J. Cell Biol. 128, 995–1002 (1995).
Mahadevan, M.S. et al. Nat. Genet. 38, 1066–1070 (2006).
Timchenko, N.A. et al. J. Biol. Chem. 279, 13129–13139 (2004).
Miller, J.W. et al. EMBO J. 19, 4439–4448 (2000).
Ebralidze, A., Wang, Y., Petkova, V., Ebralidze, K. & Junghans, R.P. Science 303, 383–387 (2004).
Charlet-B, N. et al. Mol. Cell 10, 45–53 (2002).
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Timchenko, L. Reversal of fortune. Nat Genet 38, 976–977 (2006). https://doi.org/10.1038/ng0906-976
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DOI: https://doi.org/10.1038/ng0906-976
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