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CRISPR-Cas9 editing of a TNPO3 mutation in a muscle cell model of limb-girdle muscular dystrophy type D2.
Mol Ther Nucleic Acids. 2023 Jan 11;31:324-338. doi: 10.1016/j.omtn.2023.01.004. eCollection 2023 Mar 14.
Mol Ther Nucleic Acids. 2023.
PMID: 36789274
Free PMC article.
Dystrophinopathy Phenotypes and Modifying Factors in DMD Exon 45-55 Deletion.
Poyatos-García J, Martí P, Liquori A, Muelas N, Pitarch I, Martinez-Dolz L, Rodríguez B, Gonzalez-Quereda L, Damiá M, Aller E, Selva-Gimenez M, Vilchez R, Diaz-Manera J, Alonso-Pérez J, Barcena JE, Jauregui A, Gámez J, Aladrén JA, Fernández A, Montolio M, Azorin I, Hervas D, Casasús A, Nieto M, Gallano P, Sevilla T, Vilchez JJ.
Poyatos-García J, et al. Among authors: selva gimenez m.
Ann Neurol. 2022 Nov;92(5):793-806. doi: 10.1002/ana.26461. Epub 2022 Sep 7.
Ann Neurol. 2022.
PMID: 35897138
Free PMC article.
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