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Cas9 targeting of toxic foci of RNA repeats

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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Fig. 1: Systemic administration of Cas9 targeting toxic expanded RNA repeats in mouse models of DM1.


  1. Sznajder, Ł. J. & Swanson, M. S. Int. J. Mol. Sci. 20, 3365 (2019).

    CAS  Article  Google Scholar 

  2. Thornton, C. A. Neurol. Clin. 32, 705–719 (2014).

    Article  Google Scholar 

  3. Taneja, K.L., McCurrach, M., Schalling, M., Housman, D. & Singer, R. H. J. Cell Biol. 128, 995–1002 (1995).

    CAS  Article  Google Scholar 

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

    CAS  Article  Google Scholar 

  5. Lin, X. et al. Hum. Mol. Genet. 15, 2087–2097 (2006).

    CAS  Article  Google Scholar 

  6. Wheeler, T. M., Lueck, J. D., Swanson, M. S., Dirksen, R. & Thornton, C. A. J. Clin. Invest. 117, 3952–3957 (2007).

    CAS  PubMed  PubMed Central  Google Scholar 

  7. Wheeler, T. M. et al. Nature 488, 111–115 (2012).

    CAS  Article  Google Scholar 

  8. Klein, A. F. et al. J. Clin. Invest. 129, 4739–4744 (2019).

    CAS  Article  Google Scholar 

  9. Reddy, K., Jenquin, J. R., Cleary, J. D. & Berglund, J. A. Int. J. Mol. Sci. 20, 4017 (2019).

    CAS  Article  Google Scholar 

  10. Pinto, B. S. et al. Mol. Cell 68, 479–490.e5 (2017).

    CAS  Article  Google Scholar 

  11. Bisset, D. R. et al. Hum. Mol. Genet. 24, 4971–4983 (2015).

    CAS  Article  Google Scholar 

  12. Batra, R. et al. Cell 170, 899–912.e10 (2017).

    CAS  Article  Google Scholar 

  13. Batra, R. et al. Nat. Biomed. Eng. (2020).

  14. Mankodi, A. et al. Science 289, 1769–1773 (2000).

    CAS  Article  Google Scholar 

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Correspondence to Denis Furling.

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Furling, D. Cas9 targeting of toxic foci of RNA repeats. Nat Biomed Eng 5, 130–131 (2021).

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