Molecular BiologybioRxivPreprint
Targeting Toxic Nuclear RNA Foci with CRISPR-Cas13 to Treat Myotonic Dystrophy
Pornthida Poosala, Sean R. Lindley, Kelly M. Anderson, Douglas M. Anderson
This paper demonstrates the use of CRISPR-Cas13 to target and manipulate toxic nuclear RNA foci in myotonic dystrophy type 1, offering tools for visualization and cleavage of CUG repeat RNA.
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