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Therapeutic Expression of Large Dystrophins and a CRISPR Base-Editor for Duchenne Muscular Dystrophy by Scarless RNA Trans-Splicing

Ryan H. Hsu, Claire E. Williams,Geraldine Maier,Miriam Gullo,Karen Lettieri, Cindy Alvarez, Kip J. Hermann,Lukas C. Bachmann,Samuel L. Pfaff

MOLECULAR THERAPY（2024）

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