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Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells.
Chaouch S, Mouly V, Goyenvalle A, Vulin A, Mamchaoui K, Negroni E, Di Santo J, Butler-Browne G, Torrente Y, Garcia L, Furling D. Chaouch S, et al. Among authors: mouly v. Hum Gene Ther. 2009 Jul;20(7):784-90. doi: 10.1089/hum.2008.163. Hum Gene Ther. 2009. PMID: 19358679
FUBP1: a new protagonist in splicing regulation of the DMD gene.
Miro J, Laaref AM, Rofidal V, Lagrafeuille R, Hem S, Thorel D, Méchin D, Mamchaoui K, Mouly V, Claustres M, Tuffery-Giraud S. Miro J, et al. Among authors: mouly v. Nucleic Acids Res. 2015 Feb 27;43(4):2378-89. doi: 10.1093/nar/gkv086. Epub 2015 Feb 6. Nucleic Acids Res. 2015. PMID: 25662218 Free PMC article.
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.
Klein P, Oloko M, Roth F, Montel V, Malerba A, Jarmin S, Gidaro T, Popplewell L, Perie S, Lacau St Guily J, de la Grange P, Antoniou MN, Dickson G, Butler-Browne G, Bastide B, Mouly V, Trollet C. Klein P, et al. Among authors: mouly v. Nucleic Acids Res. 2016 Dec 15;44(22):10929-10945. doi: 10.1093/nar/gkw703. Epub 2016 Aug 9. Nucleic Acids Res. 2016. PMID: 27507886 Free PMC article.
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.
Lattanzi A, Duguez S, Moiani A, Izmiryan A, Barbon E, Martin S, Mamchaoui K, Mouly V, Bernardi F, Mavilio F, Bovolenta M. Lattanzi A, et al. Among authors: mouly v. Mol Ther Nucleic Acids. 2017 Jun 16;7:11-19. doi: 10.1016/j.omtn.2017.02.004. Epub 2017 Feb 10. Mol Ther Nucleic Acids. 2017. PMID: 28624187 Free PMC article.
Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides.
Tone Y, Mamchaoui K, Tsoumpra MK, Hashimoto Y, Terada R, Maruyama R, Gait MJ, Arzumanov AA, McClorey G, Imamura M, Takeda S, Yokota T, Wood MJA, Mouly V, Aoki Y. Tone Y, et al. Among authors: mouly v. Nucleic Acid Ther. 2021 Apr;31(2):172-181. doi: 10.1089/nat.2020.0907. Epub 2021 Feb 9. Nucleic Acid Ther. 2021. PMID: 33567244 Free PMC article.
Generation of isogenic D4Z4 contracted and noncontracted immortal muscle cell clones from a mosaic patient: a cellular model for FSHD.
Krom YD, Dumonceaux J, Mamchaoui K, den Hamer B, Mariot V, Negroni E, Geng LN, Martin N, Tawil R, Tapscott SJ, van Engelen BG, Mouly V, Butler-Browne GS, van der Maarel SM. Krom YD, et al. Among authors: mouly v. Am J Pathol. 2012 Oct;181(4):1387-401. doi: 10.1016/j.ajpath.2012.07.007. Epub 2012 Aug 4. Am J Pathol. 2012. PMID: 22871573 Free PMC article.
Novel small molecules potentiate premature termination codon readthrough by aminoglycosides.
Baradaran-Heravi A, Balgi AD, Zimmerman C, Choi K, Shidmoossavee FS, Tan JS, Bergeaud C, Krause A, Flibotte S, Shimizu Y, Anderson HJ, Mouly V, Jan E, Pfeifer T, Jaquith JB, Roberge M. Baradaran-Heravi A, et al. Among authors: mouly v. Nucleic Acids Res. 2016 Aug 19;44(14):6583-98. doi: 10.1093/nar/gkw638. Epub 2016 Jul 12. Nucleic Acids Res. 2016. PMID: 27407112 Free PMC article.
215 results