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GENE THERAPY

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.

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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). https://doi.org/10.1038/s41551-021-00688-y

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