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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: mamchaoui k. Hum Gene Ther. 2009 Jul;20(7):784-90. doi: 10.1089/hum.2008.163. Hum Gene Ther. 2009. PMID: 19358679
Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.
Echigoya Y, Lim KRQ, Trieu N, Bao B, Miskew Nichols B, Vila MC, Novak JS, Hara Y, Lee J, Touznik A, Mamchaoui K, Aoki Y, Takeda S, Nagaraju K, Mouly V, Maruyama R, Duddy W, Yokota T. Echigoya Y, et al. Among authors: mamchaoui k. Mol Ther. 2017 Nov 1;25(11):2561-2572. doi: 10.1016/j.ymthe.2017.07.014. Epub 2017 Jul 28. Mol Ther. 2017. PMID: 28865998 Free PMC article.
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Hiller M, Falzarano MS, Garcia-Jimenez I, Sardone V, Verheul RC, Popplewell L, Anthony K, Ruiz-Del-Yerro E, Osman H, Goeman JJ, Mamchaoui K, Dickson G, Ferlini A, Muntoni F, Aartsma-Rus A, Arechavala-Gomeza V, Datson NA, Spitali P. Hiller M, et al. Among authors: mamchaoui k. PLoS One. 2018 Oct 2;13(10):e0204485. doi: 10.1371/journal.pone.0204485. eCollection 2018. PLoS One. 2018. PMID: 30278058 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: mamchaoui k. 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.
89 results