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Single-swap editing for the correction of common Duchenne muscular dystrophy mutations.
Chai AC, Chemello F, Li H, Nishiyama T, Chen K, Zhang Y, Sánchez-Ortiz E, Alomar A, Xu L, Liu N, Bassel-Duby R, Olson EN. Chai AC, et al. Among authors: sanchez ortiz e. Mol Ther Nucleic Acids. 2023 Apr 19;32:522-535. doi: 10.1016/j.omtn.2023.04.009. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37215149 Free PMC article.
Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy.
Nishiyama T, Zhang Y, Cui M, Li H, Sanchez-Ortiz E, McAnally JR, Tan W, Kim J, Chen K, Xu L, Bassel-Duby R, Olson EN. Nishiyama T, et al. Among authors: sanchez ortiz e. Sci Transl Med. 2022 Nov 23;14(672):eade1633. doi: 10.1126/scitranslmed.ade1633. Epub 2022 Nov 23. Sci Transl Med. 2022. PMID: 36417486 Free PMC article.
31 results