The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci of expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models of myotonic dystrophy type 1.
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References
Sznajder, Ł. J. & Swanson, M. S. Int. J. Mol. Sci. 20, 3365 (2019).
Thornton, C. A. Neurol. Clin. 32, 705–719 (2014).
Taneja, K.L., McCurrach, M., Schalling, M., Housman, D. & Singer, R. H. J. Cell Biol. 128, 995–1002 (1995).
Miller, J. W. et al. EMBO J. 19, 4439–4448 (2000).
Lin, X. et al. Hum. Mol. Genet. 15, 2087–2097 (2006).
Wheeler, T. M., Lueck, J. D., Swanson, M. S., Dirksen, R. & Thornton, C. A. J. Clin. Invest. 117, 3952–3957 (2007).
Wheeler, T. M. et al. Nature 488, 111–115 (2012).
Klein, A. F. et al. J. Clin. Invest. 129, 4739–4744 (2019).
Reddy, K., Jenquin, J. R., Cleary, J. D. & Berglund, J. A. Int. J. Mol. Sci. 20, 4017 (2019).
Pinto, B. S. et al. Mol. Cell 68, 479–490.e5 (2017).
Bisset, D. R. et al. Hum. Mol. Genet. 24, 4971–4983 (2015).
Batra, R. et al. Cell 170, 899–912.e10 (2017).
Batra, R. et al. Nat. Biomed. Eng. https://doi.org/10.1038/s41551-020-00607-7 (2020).
Mankodi, A. et al. Science 289, 1769–1773 (2000).
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Furling, D. Cas9 targeting of toxic foci of RNA repeats. Nat Biomed Eng 5, 130–131 (2021). https://doi.org/10.1038/s41551-021-00688-y
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DOI: https://doi.org/10.1038/s41551-021-00688-y